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Drug Trials Snapshots: VIZZ

Any AE 89 (61.8) 113 (78.5) 25 (34.2) -16.7 (‑26.9, ‑6.1) * 27.6 (13.6, 40.3) * 44.2 (30.8, 56.1) * Ocular hyperaemia 18 (12.5) 5 (3.5) 0 9.0 (3.0, 15.9) * 12.5 (7.3, 18.9) * 3.5 (‑1.6, 7.9) Conjunctival hyperaemia 17 (11.8) 8 (5.6) 1 (1.4) 6.2 (‑0.3, 13.2) 10.4 (3.6, 17.0) * 4.2 (‑2.2, 9.5) Instillation site erythema 6 (4.2) 3 (2.1) 0 2.1 (‑2.3, 7.0) 4.2 (‑0.9, 8.8) 2.1 (‑3.0, 6.0) Acute sinusitis 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Anisocoria 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Asthenopia 2 (1.4) 1 (0.7) 0 0.7 (‑2.6, 4.3) 1.4 (‑3.7, 4.9) 0.7 (‑4.3, 3.8) Calculus bladder 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Cataract cortical 1 (0.7) 0 1 (1.4) 0.7 (‑1.9, 3.8) ‑0.7 (‑6.7, 2.6) ‑1.4 (‑7.4, 1.3) Chalazion 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Chest pain 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Conjunctivitis allergic 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Conjunctivochalasis 1 (0.7) 0 1 (1.4) 0.7 (‑1.9, 3.8) ‑0.7 (‑6.7, 2.6) ‑1.4 (‑7.4, 1.3) Contusion 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Corneal abrasion 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Corneal dystrophy 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Corneal pigmentation 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Deep vein thrombosis 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Electric shock 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Endometrial adenocarcinoma 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Eye infection viral 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Eye irritation 2 (1.4) 1 (0.7) 0 0.7 (‑2.6, 4.3) 1.4 (‑3.7, 4.9) 0.7 (‑4.3, 3.8) Eye pain 6 (4.2) 5 (3.5) 0 0.7 (‑4.3, 5.8) 4.2 (‑0.9, 8.8) 3.5 (‑1.6, 7.9) Hand fracture 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Hypothyroidism 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Instillation site pruritus 1 (0.7) 0 1 (1.4) 0.7 (‑1.9, 3.8) ‑0.7 (‑6.7, 2.6) ‑1.4 (‑7.4, 1.3) Intraocular pressure increased 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Iris transillumination defect 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Ligament sprain 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Meibomian gland dysfunction 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Nasopharyngitis 1 (0.7) 0 1 (1.4) 0.7 (‑1.9, 3.8) ‑0.7 (‑6.7, 2.6) ‑1.4 (‑7.4, 1.3) Otorrhoea 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Pharyngitis 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Restless legs syndrome 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Retinal haemorrhage 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Rhinalgia 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Rosacea 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Skin laceration 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Urobilinogen urine increased 1 (0.7) 0 0 0.7 (‑1.9, 3.8) 0.7 (‑4.3, 3.8) 0.0 (‑5.0, 2.6) Vitreous floaters 8 (5.6) 7 (4.9) 0 0.7 (‑4.9, 6.4) 5.6 (0.4, 10.6) * 4.9 (‑0.2, 9.7) Allergic keratitis 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Back pain 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Blood glucose increased 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Cellulitis 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Dental caries 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Dysphagia 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Eye swelling 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Eyelid margin crusting 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Gingival abscess 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Hypercholesterolaemia 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Infective keratitis 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Instillation site lacrimation 0 0 1 (1.4) 0.0 (‑2.6, 2.6) ‑1.4 (‑7.4, 1.3) ‑1.4 (‑7.4, 1.3) Anxiety 2 (1.4) 2 (1.4) 0 ‑0.0 (‑3.7, 3.7) 1.4 (‑3.7, 4.9) 1.4 (‑3.7, 4.9) Conjunctival haemorrhage 1 (0.7) 1 (0.7) 0 ‑0.0 (‑3.2, 3.2) 0.7 (‑4.3, 3.8) 0.7 (‑4.3, 3.8) Conjunctival oedema 1 (0.7) 1 (0.7) 0 ‑0.0 (‑3.2, 3.2) 0.7 (‑4.3, 3.8) 0.7 (‑4.3, 3.8) Eye pruritus 2 (1.4) 2 (1.4) 2 (2.7) ‑0.0 (‑3.7, 3.7) ‑1.4 (‑8.2, 2.7) ‑1.4 (‑8.2, 2.7) Ocular discomfort 1 (0.7) 1 (0.7) 0 ‑0.0 (‑3.2, 3.2) 0.7 (‑4.3, 3.8) 0.7 (‑4.3, 3.8) Pneumonia 3 (2.1) 3 (2.1) 0 ‑0.0 (‑4.1, 4.1) 2.1 (‑3.0, 6.0) 2.1 (‑3.0, 6.0) Sinus headache 1 (0.7) 1 (0.7) 0 ‑0.0 (‑3.2, 3.2) 0.7 (‑4.3, 3.8) 0.7 (‑4.3, 3.8) Sinusitis 1 (0.7) 1 (0.7) 0 ‑0.0 (‑3.2, 3.2) 0.7 (‑4.3, 3.8) 0.7 (‑4.3, 3.8) Upper respiratory tract infection 2 (1.4) 2 (1.4) 0 ‑0.0 (‑3.7, 3.7) 1.4 (‑3.7, 4.9) 1.4 (‑3.7, 4.9) Abdominal pain upper 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Abnormal sensation in eye 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Adenoviral conjunctivitis 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Alanine aminotransferase abnormal 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Amaurosis fugax 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Aspartate aminotransferase 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Asthma 1 (0.7) 2 (1.4) 0 ‑0.7 (‑4.3, 2.6) 0.7 (‑4.3, 3.8) 1.4 (‑3.7, 4.9) Blepharitis 0 1 (0.7) 1 (1.4) ‑0.7 (‑3.8, 1.9) ‑1.4 (‑7.4, 1.3) ‑0.7 (‑6.7, 2.6) Cerebrovascular accident 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Conjunctivitis bacterial 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Constipation 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) COVID-19 3 (2.1) 4 (2.8) 1 (1.4) ‑0.7 (‑5.1, 3.5) 0.7 (‑5.4, 4.8) 1.4 (‑4.8, 5.8) COVID-19 pneumonia 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Diarrhoea 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Dysgeusia 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Ear pain 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Eye injury 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Foreign body sensation in eyes 1 (0.7) 2 (1.4) 0 ‑0.7 (‑4.3, 2.6) 0.7 (‑4.3, 3.8) 1.4 (‑3.7, 4.9) Fungal infection 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Hepatic enzyme increased 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Hypersensitivity 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Hypoxia 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Influenza 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Injection site pain 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Instillation site foreign body sensation 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Joint injury 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Loss of visual contrast sensitivity 5 (3.5) 6 (4.2) 0 ‑0.7 (‑5.8, 4.3) 3.5 (‑1.6, 7.9) 4.2 (‑0.9, 8.8) Macrocytosis 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Metapneumovirus infection 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Ovarian cyst 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Pain 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Paranasal sinus discomfort 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Prostate cancer 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Prostatitis 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Punctate keratitis 1 (0.7) 2 (1.4) 0 ‑0.7 (‑4.3, 2.6) 0.7 (‑4.3, 3.8) 1.4 (‑3.7, 4.9) Rib fracture 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Rotator cuff syndrome 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Salivary gland mass 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Skin abrasion 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Skin tightness 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Superficial injury of eye 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Taste disorder 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Urinary tract infection 3 (2.1) 4 (2.8) 1 (1.4) ‑0.7 (‑5.1, 3.5) 0.7 (‑5.4, 4.8) 1.4 (‑4.8, 5.8) Vertigo 0 1 (0.7) 0 ‑0.7 (‑3.8, 1.9) 0.0 (‑5.0, 2.6) 0.7 (‑4.3, 3.8) Eye naevus 0 2 (1.4) 0 ‑1.4 (‑4.9, 1.2) 0.0 (‑5.0, 2.6) 1.4 (‑3.7, 4.9) Fatigue 0 2 (1.4) 0 ‑1.4 (‑4.9, 1.2) 0.0 (‑5.0, 2.6) 1.4 (‑3.7, 4.9) Hypertension 0 2 (1.4) 1 (1.4) ‑1.4 (‑4.9, 1.2) ‑1.4 (‑7.4, 1.3) 0.0 (‑6.1, 3.8) Migraine 0 2 (1.4) 0 ‑1.4 (‑4.9, 1.2) 0.0 (‑5.0, 2.6) 1.4 (‑3.7, 4.9) Rhinorrhoea 0 2 (1.4) 0 ‑1.4 (‑4.9, 1.2) 0.0 (‑5.0, 2.6) 1.4 (‑3.7, 4.9) Dizziness 0 3 (2.1) 1 (1.4) ‑2.1 (‑6.0, 0.6) ‑1.4 (‑7.4, 1.3) 0.7 (‑5.4, 4.8) Lacrimation increased 2 (1.4) 5 (3.5) 0 ‑2.1 (‑6.7, 1.9) 1.4 (‑3.7, 4.9) 3.5 (‑1.6, 7.9) Visual acuity reduced 0 3 (2.1) 0 ‑2.1 (‑6.0, 0.6) 0.0 (‑5.0, 2.6) 2.1 (‑3.0, 6.0) Vitreous detachment 1 (0.7) 4 (2.8) 0 ‑2.1 (‑6.3, 1.3) 0.7 (‑4.3, 3.8) 2.8 (‑2.3, 6.9) Vomiting 0 3 (2.1) 0 ‑2.1 (‑6.0, 0.6) 0.0 (‑5.0, 2.6) 2.1 (‑3.0, 6.0) Dry eye 1 (0.7) 5 (3.5) 0 ‑2.8 (‑7.3, 0.7) 0.7 (‑4.3, 3.8) 3.5 (‑1.6, 7.9) Vision blurred 4 (2.8) 9 (6.2) 7 (9.6) ‑3.5 (‑9.0, 1.5) ‑6.8 (‑16.0, ‑0.6) * ‑3.3 (‑12.8, 3.8) Photophobia 0 6 (4.2) 0 ‑4.2 (‑8.8, ‑1.5) * 0.0 (‑5.0, 2.6) 4.2 (‑0.9, 8.8) Instillation site irritation 28 (19.4) 35 (24.3) 9 (12.3) ‑4.9 (‑14.4, 4.7) 7.1 (‑3.9, 16.6) 12.0 (0.7, 21.8) * Nausea 1 (0.7) 11 (7.6) 1 (1.4) ‑6.9 (‑12.6, ‑2.8) * ‑0.7 (‑6.7, 2.6) 6.3 (‑0.3, 12.1) Facial pain 9 (6.2) 20 (13.9) 2 (2.7) ‑7.6 (‑15.0, ‑0.7) * 3.5 (‑3.7, 9.3) 11.1 (3.3, 18.3) * Visual impairment 28 (19.4) 42 (29.2) 0 ‑9.7 (‑19.6, 0.2) 19.4 (13.8, 26.7) * 29.2 (22.3, 37.1) * Headache 21 (14.6) 51 (35.4) 5 (6.8) -20.8 (‑30.5, ‑11.0) * 7.7 (‑1.7, 15.7) 28.6 (17.9, 37.9) *

RIASTAP

STN# 125317 Proper Name: Fibrinogen Concentrate (Human) Tradename: RIASTAP Manufacturer: CSL Behring GmbH Indication: Treatment of of acute bleeding episodes in pediatric and adult patients with congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia. Product Information Package insert - RIASTAP Supporting Documents December 19, 2025 Approval Letter - RIASTAP Statistical Review - RIASTAP Clinical Review Memorandum - RIASTAP June 4, 2021 Approval Letter - RIASTAP January 16, 2009 Approval Letter - RIASTAP Summary Basis for Regulatory Action - RIASTAP (125317/0) Approval History, Letters, Reviews and Related Documents - RIASTAP

Plasminogen, Human-tvmh

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Pooled Plasma (Human), Solvent/Detergent Treated

The.gov means it’s official. Federal government websites often end in.gov or.mil. Before sharing sensitive information, make sure you're on a federal government site. The site is secure. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Prothrombin Complex Concentrate, Human-LANS

The.gov means it’s official. Federal government websites often end in.gov or.mil. Before sharing sensitive information, make sure you're on a federal government site. The site is secure. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Adverse Events and Product Deviation Guidances

Should you find a link that does not work within any Guidance document, Rule or other document posted on the FDA Web site, please try searching for the document using the document title. If you need further assistance, please go to Contact FDA. Providing Submissions in Electronic Format - Postmarketing Safety Reports for Vaccines - Guidance for Industry CBER, August 2015 Draft Guidance for Industry: Providing Submissions in Electronic Format - Postmarketing Safety Reports CDER/CBER, June 2014 Guidance for Industry: E2B(R3) Electronic Transmission of Individual Case Safety Reports Implementation Guide - Data Elements and Message Specification (version 5.01) CDER/CBER, February 2014 Guidance for Industry: Postmarketing Adverse Event Reporting for Medical Products and Dietary Supplements During an Influenza Pandemic OCET/CDER/CBER/CFSAN/CDRH, February 2012 Biological Product Deviation Reporting for Licensed Manufacturers of Biological Products Other than Blood and Blood Components; Guidance for Industry CBER/CDER, October 2006 Guidance for Industry: Adverse Reactions Section of Labeling for Human Prescription Drug and Biological Products - Content and Format CDER/CBER, January 2006 Draft Guidance for Industry: Postmarketing Safety Reporting for Human Drug and Biological Products Including Vaccines CDER/CBER, March 2001 Guidance for Industry: Postmarketing Adverse Experience Reporting for Human Drug and Licensed Biological Products: Clarification of What to Report CDER/CBER, August 1997

Handelnine Global Limited d/b/a as Navafresh Issues Nationwide Recall of Rheumacare Capsules by Virgo UAP Pharma Pvt. Ltd. (Virgo), Due to Elevated Lead Levels

Summary Company Announcement Handelnine Global Limited d/b/a Navafresh is voluntarily recalling Lot Numbers CAM040 & CALO79-N of Rheumacare Capsules by Virgo UAP Pharma Pvt. Ltd. (Virgo) to the consumer level. In test conducted by the Food and Drug Administration the product has been found to contain lead at levels up to 11,100 ppm, which are higher than permissible levels. Risk Statement: The product potentially could result in public health risk. Sustained exposure of elevated levels of lead poses increased potential toxicological risks to consumers to include cognitive impairment, depression, nausea and abdominal pain, motor incoordination, and peripheral neuropathy (loss of muscle control). Prolonged exposure has also been linked to the development of anemia. From a reproductive health standpoint, chronic lead exposure has been correlated with increased risk of miscarriage in females and reduced fertility in males. Acute toxicity of lead poisoning could lead to abdominal pain/colic, vomiting, constipation, peripheral neuropathy, and cerebral edema and encephalopathy, which can lead to seizures, coma, and death. To date, Handelnine Global Limited has received one adverse event complaint related to this recall. The product is used to reduce joint pain and is packaged in white plastic containers bearing the label of the brand Rheumacare Capsules. The affected Rheumacare Capsules lots include the following CAM040 & CALO79-N. Product was distributed nationwide via our website www.navafresh.com. Handelnine Global Limited is notifying its distributors and customers by email of the recalled product. Consumers that have product which is being recalled should stop using and discard the product immediately. A refund shall be issued to consumers who had placed orders for the product with us. Consumers with questions regarding this recall can contact Mr. Ankit Mota at Handelnine Global Limited by phone number +1 442-223-0999 or e-mail address legal@handelnine.com on Monday to Friday from 10 am to 5 pm, GMT (UK time). Consumers should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using this drug product. Adverse reactions or quality problems experienced with the use of this product may be reported to the FDA’s MedWatch Adverse Event Reporting program either online, by regular mail, or by fax. Complete and submit the report Online: www.fda.gov/medwatch/report.htm Regular Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form or submit by fax to 1-800-FDA-0178. This recall is being conducted with the knowledge of the U.S. Food and Drug Administration.

FDA approves drug to improve functional capacity and symptoms in adults with rare inherited heart condition

Action The U.S. Food and Drug Administration (FDA) approved Myqorzo (aficamten) tablets to treat adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Disease or Condition Symptomatic oHCM is an inherited condition where patients have thickened heart muscle and reduced blood flow from the left side of the heart to the rest of the body, causing symptoms such as shortness of breath, fatigue, and potentially life-threatening cardiac events. Myqorzo Effectiveness The effectiveness and safety of Myqorzo were studied in 282 adults with symptomatic oHCM randomly assigned to receive Myqorzo or placebo for 24 weeks. At the end of the study, participants receiving Myqorzo had an increase in exercise capacity measured by peak oxygen uptake compared to no change in exercise capacity among those receiving placebo. Also, 59 percent of participants receiving Myqorzo experienced an improvement in physical activity limitations (measured using the New York Heart Association Classification system) compared to 24 percent of individuals receiving placebo. Safety Information Myqorzo reduces contraction of the heart muscle which can cause heart failure. Because of the risk for heart failure, patients treated with Myqorzo must be monitored with echocardiograms, an imaging test that shows how well the heart is working. Because of the heart failure risk and need for monitoring, Myqorzo is only available through a restricted program called the Myqorzo Risk Evaluation and Mitigation Strategy (REMS). Patients must also avoid certain prescription medicines that interfere with the metabolism (breakdown) of Myqorzo. See the prescribing information for additional information on risks associated with Myqorzo. Designations Myqorzo received Orphan Drug Designation and Breakthrough Therapy Designation for this indication.

FDA approves first prescription oral medicine for iron deficiency in pediatric patients ages 10 and older

Action The U.S. Food and Drug Administration (FDA) approved Accrufer (ferric maltol) capsules to treat pediatric patients ages 10 and older with iron deficiency. Accrufer was initially approved in 2019 for adults with iron deficiency. Disease or Condition Iron deficiency is the most common cause of anemia and occurs if there is not enough iron in the blood. Without enough iron, the body cannot make sufficient hemoglobin, a protein inside red blood cells that carries oxygen throughout the body. Iron deficiency anemia can result in symptoms such as fatigue, pale skin, and dizziness. Causes can include blood loss from gastrointestinal conditions, menstruation, food malabsorption, and insufficient iron in the diet. Data Supporting Accrufer The efficacy of Accrufer to treat iron deficiency in pediatric patients ages 10-17 was assessed in the FORTIS trial (NCT05126901). The trial treated 24 patients with age-based dosing of Accrufer twice daily, and showed a clinically meaningful average increase in hemoglobin of 1.1 g/dL at Week 12. This, on average, would be the expected increase in hemoglobin with one blood transfusion. Safety Information Patients should not take Accrufer if they are allergic to Accrufer or any of its excipients, have hemochromatosis and other iron overload syndromes, or receive repeated blood transfusions. In addition, patients should avoid Accrufer if they are experiencing an inflammatory bowel disease flare and should be aware of the risks of iron overload. The most common side effects of Accrufer are flatulence, diarrhea, constipation, discolored feces, abdominal pain, nausea, vomiting, and abdominal discomfort/distension.

CDER Leadership Bios

Amy Comstock Rick, J.D. Associate Director for Rare Disease Strategy Amy Comstock Rick, J.D., is CDER’s Associate Director for Rare Disease Strategy and the Director of Strategic Coalitions for FDA’s Rare Disease Innovation Hub (the Hub). She serves in a cross-cutting role across FDA’s Center for Drug Evaluations and Research (CDER) and Center for Biologics Evaluation and Research (CBER) to facilitate implementation of the Hub. More about Amy Comstock Rick, J.D. Theresa M. Mullin, Ph.D. Associate Director for Strategic Initiatives Theresa Mullin, Ph.D., serves as CDER’s Associate Director for Strategic Initiatives. She oversees areas of strategic interest to external stakeholders. She leads the Patient-Focused Drug Development (PFDD) initiative, which includes work related to the FDA Reauthorization Act (FDARA) and implementation of the 21st Century Cures Act. She also leads CDER’s International Program. More about Theresa M. Mullin Ph.D. Patrick Raulerson, J.D. Director, Office of Regulatory Policy As Director of CDER’s Office of Regulatory Policy (ORP), Patrick Raulerson, J.D., provides leadership and guidance to the Center regarding policies and procedures related to the regulation of human drugs. He is also the chair of CDER’s Strategic Policy Council. More about Patrick Raulerson, J.D. Mary T. Thanh Hai, M.D. Director, Office of New Drugs Mary Thanh Hai, M.D. serves as the Director of the Office of New Drugs (OND) in the Center for Drug Evaluation and research. OND oversees the clinical and nonclinical investigations supporting drug development programs for prescription drugs and therapeutic biologics and non-prescription drugs. More about Mary T. Thanh Hai, M.D. ShaAvhree Buckman-Garner, M.D., Ph.D., FAAP Director, Office of Translational Sciences ShaAvhrée Buckman-Garner, M.D., Ph.D., FAAP is the Director of CDER's Office of Translational Sciences (OTS). OTS is comprised of the Office of Biostatistics, Office of Clinical Pharmacology, Office of Computational Science, Office of Study Integrity and Surveillance, provides oversight to CDER research involving human subjects, the CDER ORISE Program, as well as CDER regulatory science research. OTS is responsible for providing coordination for Critical Path initiatives across CDER in partnership with individual CDER offices. More about ShaAvhree Buckman-Garner MD, Ph.D., FAAP Carter Beach, J.D. Director, Office of Executive Programs As Director of the Office of Executive Programs (OEP), Carter Beach oversees a variety of programs for the Center for Drug Evaluation and Research (CDER), including executive operations, legislative affairs, project management, learning and organizational development, the scientific advisory committee program, product jurisdiction and combination products, and formal dispute resolution and ombudsman services. More about Carter Beach, R.Ph. Gerald J. Dal Pan, M.D., M.H.S. Director, Office of Surveillance and Epidemiology Gerald J. Dal Pan became the Director of the Office of Surveillance and Epidemiology (known then as the Office of Drug Safety) in November 2005. Before that, he was the Director of the Division of Surveillance, Research, and Communication Support. More about Gerald J. Pan, M.D., M.H.S. M. Khair ElZarrad, Ph.D., M.P.H. Director, Office of Medical Policy M. Khair ElZarrad, Ph.D., M.P.H., is the Director of the Office of Medical Policy (OMP) in FDA’s Center for Drug Evaluation and Research (CDER). He has served as the Deputy Director of OMP since 2017. More about M. Khair ElZarrad, Ph.D., M.P.H. Eric Stone, M.B.A. Director, Office of Management Eric Stone is Director of CDER's Office of Management. He also serves as the CDER Executive Officer and CDER’s Associate Director for Management. He leads the center’s administrative management operations, which includes more than 5,000 employees and an annual budget of more than $1 billion, comprised of a mix of congressional appropriations and multiple user fee programs. Under Mr. Stone’s leadership, the Office of Management staff manages all aspects of: ethics management, human capital management, financial management (including budget formulation, user-fee collection, and budget execution), acquisition management, employee safety, and facilities management. More about Eric Stone, M.B.A. Sridhar Mantha, M.B.A., M.S., B.Tech. Director, Office of Strategic Programs Sridhar (Sri) Mantha, M.B.A., M.S., B.Tech., serves as Director of CDER’s Office of Strategic Programs (OSP). OSP plays a lead role in many of the center’s strategic initiatives and modernization efforts, including development of benefit-risk and other decision support tools, data standardization, data governance, lean process management, program analysis, enterprise informatics, resource capacity planning, and major user fee negotiations. OSP leads implementation of CDER’s business informatics governance function in support of business modernization objectives. More about Sridhar Mantha, M.B.A., M.S., B.Tech. Jill Furman, J.D. Director, Office of Compliance Jill Furman, J.D., serves as the Director of the Office of Compliance in the Center for Drug Evaluation and Research (CDER). The Office of Compliance is charged with protecting the public from poor quality, unsafe, and ineffective drug products through proactive compliance strategies and risk-based enforcement actions. More about Jill Furman, J.D.

Update on the Safety of Andexxa by AstraZeneca: FDA Safety Communication

AUDIENCE: Patient, Health Care Professional, Pharmacy, Hematology ISSUE: Since approval, the FDA has received postmarketing safety data on thromboembolic events, including serious and fatal outcomes, in patients treated with Andexxa (coagulation factor Xa (recombinant), inactivated-zhzo). Based on available data, the serious risks including the increase in thromboembolic events are such that the FDA considers the risks of the product to outweigh its benefits. The FDA has communicated this position to AstraZeneca, and the company has submitted a request to voluntarily withdraw the BLA for the product for commercial reasons. Additionally, the company has confirmed that it will end U.S. commercial sales today, December 22, 2025. Andexxa will no longer be manufactured for or sold in the U.S. by AstraZeneca after December 22, 2025. Continuous monitoring and assessment of the safety of all biological products, including Andexxa, is an FDA priority, and we remain committed to informing the public when we learn new information about these products. BACKGROUND: The FDA initially granted accelerated approval (AA) of Andexxa, a recombinant modified human factor Xa (FXa) protein, in 2018, indicated for patients treated with rivaroxaban or apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. Initial approval included a Boxed Warning for thromboembolic risks. AA was granted based on the change from baseline in anti-activated FXa (anti-FXa) activity in healthy volunteers, as a surrogate endpoint reasonably likely to predict clinical benefit. At the time of AA of Andexxa, AstraZeneca (Applicant) was subject to a requirement to conduct a randomized controlled trial (NCT03661528) to verify the clinical benefit of Andexxa among patients with intracerebral hemorrhage following treatment with rivaroxaban or apixaban. On January 31, 2024, the Applicant submitted a supplemental Biologics Licensing Application (sBLA) for Andexxa with the results of the ANNEXA-I trial to fulfill this requirement. The FDA convened a meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee (AC) on November 21, 20241, to discuss the results of the ANNEXA-I trial. The major safety findings discussed at the AC meeting included a doubling of the rate of thromboses and thrombosis-related deaths at Day 30 in the Andexxa arm compared with usual care (UC): Increased risk of thrombosis: ANNEXA-I demonstrated an increased incidence of thrombosis (14.6% versus 6.9%) and thrombosis-related deaths at Day 30 (2.5% versus 0.9%) in the Andexxa arm compared with the UC arm, respectively. Death related to thrombotic events through 30 days occurred in 6 patients (2.5%) in the Andexxa arm compared with 2 patients (0.9%) in the usual care control arm. The FDA will continue working with AstraZeneca to keep providers and the public informed as AstraZeneca prepares to end sale of Andexxa. 1Cellular, Tissue, and Gene Therapies Advisory Committee November 21, 2024 Meeting Announcement

Coagulation Factors

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Formal Dispute Resolution and Administrative Hearings of Final Administrative Orders Under Section 505G of the Federal Food, Drug, and Cosmetic Act | FDA

Docket Number: FDA-2023-D-2204 Issued by: Guidance Issuing Office Center for Drug Evaluation and Research The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Formal Dispute Resolution and Administrative Hearings of Final Administrative Orders Under Section 505G of the Federal Food, Drug, and Cosmetic Act.” This guidance provides recommendations for industry and review staff on the formal dispute resolution (FDR) and administrative hearings procedures for resolving scientific and/or medical disputes between the Center for Drug Evaluation and Research (CDER) and requestors and sponsors of drugs that will be subject to a final administrative order under section 505G of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (“Final Order”). This guidance finalizes the draft guidance of the same title issued on June 23, 2023. Submit Comments Submit Comments Online You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5)) If unable to submit comments online, please mail written comments to: Dockets Management Food and Drug Administration 5630 Fishers Lane, Rm 1061 Rockville, MD 20852 All written comments should be identified with this document's docket number: FDA-2023-D-2204.

Biosimilars Research: Awards

The bioassay initiative: enhanced biosimilar testing capabilitiesOPQ/OPQRMay 20232-3 yearsResearch Priority C: Define best-practices for assessing and reporting quality attributesEnhancing CDER bioassay capabilities to additional attributes to provide definition, standardization, and harmonization of expectations for assessing and reporting CQAs, while reducing inconsistencies and unnecessary testingImpact 1: Increase reliance on analytical data in a demonstration of biosimilarity 2025 Annual Report 2024 Annual Report Establishment of a Feasible Method to Quantify Glycoforms of Human IgG1 mAb Drugs and their Biosimilars in Culture Media as a Component of Process Analytic Technology OPQ/OPQRMay 20232 yearsResearch Priority B: Explore how modernization of analytical technologies could better and/or more efficiently detect relevant quality attributesDevelop mouse monoclonal antibodies against human non-glycosylated and glycosylated IgG1, and establish a Biolayer Interferometry (BLI) method to quantify the major glycoforms of human IgG1 mAb drugsImpact 1: Increase reliance on analytical data in a demonstration of biosimilarity 2025 Annual Report 2024 Annual Report Develop acceptance parameters and standards for the Innate Immune Response Modulating Impurities (IIRMI) assays in the Biosimilar spaceOPQ/OPQRApril 20233 yearsResearch Priority D: Develop alternatives to comparative clinical immunogenicity assessment(s)Develop in vitro assays that can be used to reduce/replace clinical trials to assess immunogenicity risk of biosimilarsImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects 2025 Annual Report 2024 Annual Report One-Pot Glycan - a chemoenzymatic method for simultaneous profiling and comparison of N- and O-glycansOPQ/OPQRApril 20232-3 yearsResearch Priority B: Explore how modernization of analytical technologies could better and/or more efficiently detect relevant quality attributes​Establish and validate a chemoenzymatic method for simultaneous profiling and comparison N-and O-glycans from purified proteins and protein drugs in a One-Pot formatImpact 1: Increase reliance on analytical data in a demonstration of biosimilarity 2025 Annual Report 2024 Annual Report Model development and verification to evaluate minimum stability data required for biosimilar submissionsOPQ/OPQRSeptember 20233 yearsResearch Priority B: Characterize relationships between product quality attributes with clinical outcomesDetermine the minimum amount of stability data required to accurately predict long term stability and support biosimilar product’s shelf-lifeImpact 1: Increase reliance on analytical data in a demonstration of biosimilarity 2025 Annual Report 2024 Annual Report Landscape Assessment of Biosimilar SubmissionsOTS/OCP/DARSMay 20231 yearResearch Priority A: Characterize relationships between product quality attributes with clinical outcomesConduct a landscape analysis to determine how FDA can answer questions about whether differences in analytical assessments in biosimilar development programs do or do not corelate with clinical dataImpact 1: Increase reliance on analytical data in a demonstration of biosimilarity2024 Annual ReportAddressing fundamental issues for in vitro immunogenicity testingOTS/OCP/DARSMay 20231 yearResearch Priority D: Develop alternatives to comparative clinical immunogenicity assessment(s)Validate methods for in vitro immunogenicity testing that could be used by industry to reduce/eliminate the need for clinical trials assessing immunogenicity for biosimilar drug productsImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects 2025 Annual Report 2024 Annual Report Validation of a non-clinical immunogenicity modelOTS/OCP/DARSApril 20231 yearResearch Priority D: Develop alternatives to comparative clinical immunogenicity assessment(s)Evaluate the ability of humanized mice to serve as a non-clinical immunogenicity model by evaluating several biological drug products (with clinically moderate to high immunogenicity) alone or in combination in the two most commonly used humanized mouse modelsImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects 2025 Annual Report 2024 Annual Report Production & optimization of humanized miceOTS/OCP/DARSSeptember 20231 yearResearch Priority D: Develop alternatives to comparative clinical immunogenicity assessment(s)Use an established protocol for producing immune humanized mice to generate a cohort for answering regulatory questions related to pharmacokinetics, pharmacodynamics, immunogenicity and adverse events that have not been successfully addressed with other nonclinical modelsImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects 2025 Annual Report 2024 Annual Report Critical Factors for Standardization and Accuracy of PK Assays of PEGylated Biosimilars OTS/OCP/DARSSeptember 20231 year Research Priority E: Define development approaches that will increase feasibility and/ or likelihood of successProvide guidance/best practices to industry for evaluating pharmacokinetics (PK) associated with biosimilars that are conjugated to polyethylene glycol (PEG)Impact 2: Develop alternatives to and/or reduce the size of studies involving human subjects​ 2025 Annual Report 2024 Annual Report Translating Clinical Pharmacology Biosimilar Research Findings into Best Practices for Industry and FDA Review StaffOTS/OCP/DARSSeptember 20231 yearResearch Priority E: Define development approaches that will increase feasibility and/ or likelihood of successCloseout of bioanalytical and omics related project activities from the ‘Pharmacodynamic Biomarkers for Biosimilar Approval’ projectImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects2024 Annual ReportEvidence-based approach to the design of clinical pharmacology studiesOTS/OCPSeptember 20232 yearsResearch Priority E: Define development approaches that will increase feasibility and/ or likelihood of successIncrease the efficiency of biosimilar development programs by leveraging clinical pharmacology studiesImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects​ 2025 Annual Report 2024 Annual Report Assessment of Immunogenicity in Biosimilars a Systematic ReviewOND/OTBBOctober 20252 yearsResearch Priority A: Characterize relationships between product quality attributes with clinical outcomesTo examine immunogenicity data reviewed by the FDA as included in submissions for approved biosimilars and the corresponding pharmacokinetics (PK) and safety data in a definitive manner using a PRISMA based Systematic Review to determine if associations exist antidrug antibody status and clinical safety events such as hypersensitivityImpact 1: Increase reliance on analytical data in a demonstration of biosimilarityThe annual report for this project will be shared once available.Assessment of the performance of MAM vs conventional QC methods for evaluation of Product Quality Attributes of adalimumab and etanerceptU.S. PHARMACOPEIAAugust 20222-3 yearsResearch Priority B: Explore how modernization of analytical technologies could better and/or more efficiently detect relevant quality attributesTo assess the performance of the MS-based MAM versus conventional QC methods to identify changes in PQAs upon forced degradation and to correlate changes in those PQAs with bioactivity, binding affinity, and structure.Impact 1: Increase reliance on analytical data in a demonstration of biosimilarity​ 2023 Annual Report 2024 Annual Report Platform for reliable characterization and evaluation of comparability of biosimilar drug products in lyophilized and liquid formulations​NATIONAL INSTITUTE FOR PHARM TECH/EDUCAugust 20222-3 yearsResearch Priority C: Define best-practices for assessing and reporting quality attributesTo develop a platform for reliable characterization and evaluation of comparability of biosimilar drug products in lyophilized and liquid formulationsImpact 1: Increase reliance on analytical data in a demonstration of biosimilarity 2023 Annual Report 2024 Annual Report Systematic Analytical Characterization of Innovator and Biosimilar Products with the Focus on Post-translational Modifications​UNIVERSITY OF MICHIGAN AT ANN ARBORAugust 20222-3 yearsResearch Priority C: Define best-practices for assessing and reporting quality attributesTo develop novel methodologies to evaluate structural similarities and differences between innovator and multiple biosimilar products and apply them across different protein drug classes. The proposed work will establish the correlation between post-translation modification differences, receptor binding, antibody dependent cellular cytotoxicity, aggregation propensity, immunogenicity and clinical efficacy to aid approvals of biosimilar and interchangeable protein products.Impact 1: Increase reliance on analytical data in a demonstration of biosimilarity 2023 Annual Report 2024 Annual Report ISPRI-HCP: CHO protein impurity immunogenicity risk prediction for improving biosimilar product development and assessing product interchangeability​EPIVAX, INC.August 20222-3 yearsResearch Priority D: Develop alternatives to comparative clinical immunogenicity assessment(s)To improve the ISPRI-HCP immunogenicity risk prediction model for biosimilar product development and assessing product interchangeabilityImpact 2: Develop alternatives to and/or reduce the size of studies involving human subjects 2023 Annual Report 2024 Annual Report Bridging the Gap: Using Foreign Real-World Data to Inform Interchangeable Biosimilar Approvals​ACADEMY OF MANAGED CARE PHARMACY, INC.September 20232-3 yearsResearch Priority D: Develop alternatives to comparative clinical immunogenicity assessment(s)To support research projects that enhance biosimilar and interchangeable biological product development and regulatory science.Impact 2: Develop alternatives to and/or reduce the size of studies involving human subjects 2025 Annual Report 2024 Annual Report

Standards Development for Regenerative Medicine Therapies

Section 3036 of the 21st Century Cures Act refers to the development of standards for regenerative medicine therapies, including products designated Regenerative Medicine Advanced Therapies (RMATs). In consultation with the National Institute of Standards and Technology (NIST) and stakeholders, FDA is facilitating efforts to coordinate the development of standards for regenerative medicine therapies. Beginning in 2017, FDA has awarded an annual contract to Nexight Group and the Standards Coordinating Body (SCB) to coordinate community efforts toward the development of standards for regenerative medicine therapies. Under these contracts, Nexight has conducted public workshops and webinars to develop processes to enable identification of needed standards and facilitated the development of standards that have a high impact on the quality and safety of regenerative medicine therapies. In addition, SCB maintains the Regenerative Medicine Standards Portal (an interactive database that displays standards applicable to regenerative medicine therapies and the status of these standards (identified as needed, under development, or available). In October 2023, FDA published “Guidance for Industry: Voluntary Consensus Standards Recognitions Program for Regenerative Medicine Therapies”. This guidance describes a standards recognition program (SRP-RMT) at FDA’s Center for Biologics Evaluation and Research (CBER) designed to identify and recognize Voluntary Consensus Standards (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products regulated by CBER when such standards are appropriate. CBER encourages the use of appropriate standards in the development of CBER-regulated products. The use of recognized VCS can assist stakeholders in meeting regulatory requirements more efficiently and increasing regulatory predictability for RMT products. CBER is posting a list of recognized standards on this page as stated in the guidance. CBER Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies Last updated on December 13, 2024