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 neoadjuvant 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 <12 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)
(Avastin biosimilar)
Biocon Certain oncology indications CRL (facility inspection issues)
Source: Evaluate Pharma & company releases.

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