US FDA approval and panel tracker: May 2023

 Last month the FDA granted approval for the first and second ever respiratory syncytial virus vaccines, from GSK and Pfizer respectively. Both companies have committed to conducting postmarketing studies to assess signals of Guillain-Barré syndrome and other immune-mediated demyelinating conditions. Another big event for the jabs is looming, with the CDC's advisory committee on immunization practices due to meet on 21 June to discuss how the vaccines should be used. Both companies anticipate launch before the upcoming RSV season. Separately Pfizer also gained a positive adcom for Abrysvo’s use in very young children, via maternal vaccination. Sarepta had its ups and downs in May: after a favourable adcom for its DMD gene therapy contender SRP-9001, the FDA decided to postpone the final decision until 22 June. An accelerated approval is now expected to be restricted to patients aged 4-5 years old. And Intercept's Nash dream came crashing down again as an adcom voted against obeticholic acid, causing shares to sink to a record low. The final nail in the coffin, another CRL, is expected later this month.

This snippet has been updated to include Pfizer's postmarketing plans for its RSV vaccine in older adults.

Notable first-time US approval decisions in May 2023
Project	Company	Indication(s)	2028e SBI ($m)	Outcome
SRP-9001 (delandistrogene moxeparvovec)	Sarepta	Ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene	2,036	Delayed from 29 May until 22 June, positive adcom
Veozah (fezolinetant)	Astellas	Moderate-to-severe vasomotor symptoms associated with menopause	1,904	Approved
Arexvy	GSK	Prevention of lower respiratory tract disease caused by RSV in adults ≥60 years	1,729	Approved
Abrysvo	Pfizer	Prevention of lower respiratory tract disease caused by RSV in adults ≥60 years	1,306*	Approved
Epkinly (epcoritamab, DuoBody-CD3xCD20)	Genmab/Abbvie	Relapsed/refractory large B-cell lymphoma after ≥2 lines of systemic therapy	1,187	Approved (accelerated)
Bimzelx	UCB	Plaque psoriasis	718	FDA issued a Form 438 relating to manufacturing deficiencies
Vyjuvek (B-Vec, beremagene geperpavec)	Krystal	Dystrophic epidermolysis bullosa	636	Approved
Inpefa (sotagliflozin)	Lexicon	Heart failure (HFrEF & HFpEF pts with/out diabetes)	333	Approved
Alhemo (concizumab)	Novo Nordisk	Haemophilia A and B with inhibitors	198	CRL (additional information requested)
Xacduro (sulbactam-durlobactam, Sul-Dur)	Innoviva (Entasis)	Hospital‐acquired and ventilator‐associated bacterial pneumonia caused by Acinetobacter baumannii‐calcoaceticus complex in adults	82	Approved
Mydcombi	Eyenovia	Drug-device combination for in-office pupil dilation (mydriasis)	54	Approved
Elfabrio (pegunigalsidase alfa, PRX-102)	Chiesi/Protalix	Fabry disease	-	Approved
Trastuzumab duocarmazine (SYD985)	Byondis	Her2-positive unresectable breast cancer	-	CRL (additional information requested)
Brixadi	Camurus	Opioid use disorder	-	Approved
Anktiva (N-803)	Immunitybio	BCG-unresponsive non-muscle-invasive bladder cancer	-	CRL (manufacturing deficiencies and CMC problems)
Miebo (NOV03)	Bausch & Lomb	Dry eye disease	-	Approved
Posluma (flotufolastat F 18/ 18F-rhPSMA-7.3)	Blue Earth Diagnostics/ Bracco	PSMA-targeted PET imaging agent for prostate cancer	-	Approved
*Older adults and maternal setting not split out. SBI: sales by indication. Sources: Evaluate Pharma & company releases.

 

Advisory committee meetings in May 2023
Project	Company	Indication	2028e SBI ($m)	Outcome
SRP-9001 (delandistrogene moxeparvovec)	Sarepta	Ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene	2,036	8-6 in favour
Abrysvo	Pfizer	Prevention of (severe) lower respiratory tract disease caused by RSV in infants from birth through 6 months by active immunisation of pregnant individuals	1,306*	In favour, 14-0 on effectiveness and 10-4 on safety
Ocaliva (obeticholic acid)	Intercept	Pre-cirrhotic liver fibrosis due to Nash	663	12-2 against (benefits do not outweigh the risks) 15-1 vote to defer until outcomes data are available
Epinephrine nasal spray (Neffy)	ARS Pharmaceuticals	Emergency treatment of allergic reactions (type I) including anaphylaxis in adults and children ≥30 kg	-	16-6 in favour (adults), 17-5 in favour (children)
Opill	Perrigo	Daily oral contraceptive (progestin-only)	-	17-0 in favour of making Opill available for OTC use
*Older adults and maternal setting not split out. SBI: sales by indication. Sources: FDA adcom calendar, Evaluate Pharma & company releases.

 

Supplementary and other notable approval decisions in May 2023
Product	Company	Indication (clinical trial)	Outcome
Kalydeco	Vertex	Cystic fibrosis in children from 1 month to <4 months of age	Approved
Paxlovid	Pfizer	Patients who are at high risk of progression to severe disease from Covid-19 (additional analyses of Epic-HR and Epic-SR trials)	Approved
Rexulti	Otsuka/ Lundbeck	Agitation associated with Alzheimer’s dementia	Approved
Ayvakit	Blueprint	Indolent systemic mastocytosis (Pioneer)	Approved
Opvee (OPNT003 nasal nalmefene)	Indivior (Opiant)	Opioid overdose (NCT04759768, NCT05219669, NCT04828005)	Approved
Rinvoq	Abbvie	Adult patients with moderately to severely active Crohn's disease (U-Exceed, U-Excel, U-Endure)	Approved
Farxiga	Astrazeneca	Heart failure with preserved ejection fraction (Deliver)	Approved
Yuflyma (high concentration Humira biosimilar)	Celltrion	RA, juvenile idiopathic arthritis, PsA, ankylosing spondylitis, Crohn’s disease, UC, plaque psoriasis and hidradenitis suppurativa	Approved
RA: rheumatoid arthritis; PsA: psoriatic arthritis; UC: ulcerative colitis. Source: Evaluate Pharma & company releases. https://www.evaluate.com/vantage/articles/insights/nme-approvals-snippets/us-fda-approval-and-panel-tracker-may-2023