US FDA approval tracker: August

 Fibrogen’s roxadustat received an expected FDA knockback last month after a negative panel in July. The FDA requested another study, but in the EU the outcome was wildly different, and full approval was granted eight days after the US disappointment. Two other US complete response letters were handed out, one for Spectrum’s Rolontis, owing to manufacturing issues, and the other for Sesen’s Vicineum. The regulators requested a whole new confirmatory trial for Sesen’s bladder cancer project, pushing a resubmission out to 2023. However, it was not all bad news last month as several approval decisions pegged for September came early. The most notable involved Ascendis’s growth hormone Skytofa, and Bristol Myers Squibb’s Opdivo for adjuvant treatment of high-risk urothelial carcinoma; the latter had been filed for muscle-invasive disease, based on the Checkmate-274 study, but the approved label makes no mention of this stipulation (Go or no go? A first for Nefecon, August 27, 2021).

Notable first-time US approval decisions in August
Project	Company	Indication(s)	2026e sales by indication ($m)	Outcome
Comirnaty	Pfizer/Biontech	Prevention of Covid-19 (aged 16 and older)	2,819	Full approval (still under EUA for ages 12-15)
Evrenzo (roxadustat)	Fibrogen/Astrazeneca/ Astellas	Anaemia in patients with chronic kidney disease on/not on dialysis	1,666	CRL (additional trial needed)
Skytrofa (Transcon hGH/ lonapegsomatropin)	Ascendis	Paediatric growth hormone deficiency	1,371	Approved (~1mth early)
AXS-05	Axsome	Major depressive disorder	1,277*	Delayed (no new date given)
Welireg (MK-6482/belzutifan)	Merck & Co	Von Hippel-Lindau disease-associated renal cancer	386	Approved (~1mth early)
Nexviazyme (avalglucosidase alfa)	Sanofi	Pompe disease	366	Approved
Korsuva injection	Cara/Vifor	Pruritis in haemodialysis patients	358	Approved
Rolontis	Spectrum	Neutropenia  in patients receiving myelosuppressive anti-cancer drugs	330	CRL (manufacturing, reinspection needed)
Vicineum	Sesen Bio	High risk BCG-unresponsive non-muscle invasive bladder cancer	292	CRL (CMC & new trial)
TicoVac	Pfizer	Tick-borne encephalitis	218	Approved
Topiramate oral solution	Eton	Tonic-clonic seizures, partial-onset seizures, and as preventative treatment of migraine	-	Extended to Nov 6
Epsolay	Sol-Gel	Papulopustular rosacea	-	No decision yet
*SBI not split out by depression type. Source: Evaluate Pharma & company releases.

 

Supplementary and other notable approval decisions in August
Product	Company	Indication (clinical trial)	Outcome
Jemperli	GSK/Anaptysbio	Adult patients with MMR-deficient recurrent or advanced solid tumours (Garnet)	Approved (accelerated)
Jardiance	Lilly/Boehringer	Heart failure with reduced ejection fraction (Emperor-Reduced)	Approved (~3wks early)
Opdivo	Bristol Myers Squibb	Adjuvant treatment for patients with high-risk urothelial carcinoma (Checkmate-274)	Approved (2wks early)
Xarelto	J&J	Peripheral artery disease patients post lower-extremity revascularisation (Voyager PAD)	Approved
Tibsovo	Servier	Previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation (ClarIDHy)	Approved
Xywav	Jazz	Adult patients with idiopathic hypersomnia (NCT03533114)	Approved
Keytruda + Lenvima	Merck/Eisai	1L renal cell carcinoma (Clear/Keynote-581/Study 307)	Approved
Source: Evaluate Pharma & company releases.

 

Voluntarily withdrawn accelerated approvals
Product	Company	Withdrawn indication (clinical trials)
Istodax	Bristol Myers Squibb	2L peripheral T-cell lymphoma
Tecentriq (+ Abraxane)	Roche	PD-L1-positive, 1L triple-negative breast cancer (Impassion-130, Impassion-131)
Source: company releases. https://www.evaluate.com/vantage/articles/news/snippets/us-fda-approval-tracker-august-0