US FDA approval tracker: February 2023

 There were several high-profile approvals last month, including for Apellis’s Syfovre, given a broad label and a flexible dosing regimen in geographic atrophy. However, Apellis might not have the market to itself for long as Iveric Bio’s Zimura has a Pdufa date in August. Reata gained a surprise greenlight for its Friedreich's ataxia therapy Skyclarys, despite a complex regulatory path. However, Cytokinetics received a knockback for its heart failure project omecamtiv after an earlier negative adcom. The company has no plans to run another study, leaving it to focus on aficamten in obstructive hypertrophic cardiomyopathy, with phase 3 data due later this year. Lastly, the outcome of an adcom for Pfizer’s adult respiratory syncytial virus (RSV) vaccine Abrysvo was less clear-cut than might have been expected, with a 7 to 4 vote in favour. Panellists highlighted incidences of Guillain-Barré Syndrome, and post-marketing studies are expected to evaluate this risk. Stifel analysts note that GSK, whose own vaccine Arexvy is up before a panel today, has agreed with the FDA to monitor the syndrome as part of its pharmacovigilance programme.

Notable first-time US approval decisions in February
Project	Company	Indication(s)	2028e SBI ($m)	Outcome
Syfovre (intravitreal pegcetacoplan)	Apellis	Geographic atrophy secondary to age-related macular degeneration	2,310	Approved
Altuviiio (efanesoctocog alfa)	Sanofi	Ultra-long-acting FVIII therapy for haemophilia A	879	Approved
Skyclarys (omaveloxolone)	Reata	Friedreich's ataxia in patients aged 16 and older	838	Approved
Fezolinetant	Astellas	Moderate to severe vasomotor symptoms associated with menopause	699	Extended to 22 May (more time to review)
Filspari (sparsentan)	Travere	IgA nephropathy	412	Approved (accelerated)
Jesduvroq (Duvroq, daprodustat)	GSK/Kyowa Kirin	Anaemia due to CKD in adults on/not on dialysis	355	Approved in dialysis patients, CRL in non-dialysis
Omecamtiv mecarbil	Cytokinetics	Reduce the risk of CV death and heart failure events in patients with symptomatic chronic HFrEF	326	CRL (additional trial required)
Lamzede (velmanase alfa)	Chiesi	Alpha-mannosidosis	-	Approved
SBI: sales by indication. Source: Evaluate Pharma & company releases.

 

 

Advisory committee meetings in February
Project	Company	Indication	2028e SBI ($m)	Outcome
Abrysvo (RSVpreF)	Pfizer	Prevention of acute respiratory disease and lower respiratory tract disease caused by RSV in adults ≥60 years	1,216	7 to 4 in favour (note GSK's vaccine Arexvy has an adcom on March 1)
Narcan nasal spray (4mg/0.1ml)	Emergent Biosolutions	Nonprescription treatment of known or suspected opioid overdose	175	Unanimously in favour
Jemperli	GSK	Monotherapy in early treatment of MMRd/MSI-high rectal cancer	-	8 to 5 in favour of data from two single-arm trials being sufficient to characterise benefits and risks (Jemperli moves towards adjuvant rectal cancer at last)
RSV: respiratory syncytial virus. Source: Evaluate Pharma, company releases & FDA adcom calendar.

 

Supplementary and other notable approval decisions in February
Product	Company	Indication (clinical trial)	Outcome
Empaveli	Apellis	PNH, treatment-naive patients, and long-term safety and efficacy data (Prince, Pegasus)	Approved
Trodelvy	Gilead	Pretreated HR+/Her2-metastatic breast cancer (Tropics-02)	Approved
Eylea	Regeneron/ Sanofi	Retinopathy of prematurity in preterm infants (Firefleye, Butterfleye)	Approved
Tezspire	Astrazeneca/ Amgen	Self-admin in a pre-filled, single-use pen for patients ≥12 years with severe asthma (Path-Home, Path-Bridge)	Approved
Kevzara	Sanofi/ Regeneron	Polymyalgia rheumatica (Saphyr)	Approved
Takhzyro	Takeda	Hereditary angioedema attacks in children 2 to <6 years (Help, Spring)	Approved
Jemperli	GSK	MMRd endometrial cancer, progressed on or following a prior platinum-containing regimen (long-term outcomes from Garnet)	Full approval
Cibinqo	Pfizer	Adolescents with refractory, moderate-to-severe atopic dermatitis whose disease is not adequately controlled with other systemic drug products (Jade Teen)	Approved
Austedo XR	Teva	Once-daily formulation for tardive dyskinesia and chorea associated with Huntington’s disease	Approved
Voquezna (Vonaprazan, Takecab)	Phathom	Erosive oesophagitis (Phalcon-ee) and H pylori	CRLs, stability data requested owing to impurity previously found in product, launch of Voquezna Dual and Triple Pak on hold (H pylori indication)
Krabeva/MYL-1402O (Avastin biosimilar)	Biocon	Certain oncology indications	CRL (facility inspection issues)
Source: Evaluate Pharma & company releases. https://www.evaluate.com/vantage/articles/insights/nme-approvals-snippets/us-fda-approval-tracker-february-2023