US FDA approval tracker: August 2021

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/
Anaemia in patients with chronic kidney disease on/not on dialysis 1,666 CRL
(additional trial needed)
Skytrofa (Transcon hGH/
Ascendis Paediatric growth hormone deficiency 1,371 Approved (~1mth early)
AXS-05 Axsome Major depressive disorder 1,277* Delayed (no new date given)
Merck & Co Von Hippel-Lindau disease-associated renal cancer 386 Approved (~1mth early)
(avalglucosidase alfa)
Sanofi Pompe disease 366 Approved
Korsuva injection Cara/Vifor Pruritus 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.

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