US FDA approval tracker: April

Last month the US regulator tightened its grip over PI3K inhibitors by calling for randomised data instead of single-arm clinical studies. Ahead of an advisory committee meeting on the subject, TG Therapeutics pulled its own PI3K asset Ukoniq from sale and withdrew the filing of its combination product, U2. Elsewhere both Alnylam and Merck had Pdufa dates extended. For the latter, its 15-valent pneumococcal vaccine Vaxneuvance was heading for a decision in infants and children, a larger market than its already approved adult label. Despite the three-month delay Merck is still ahead of Pfizer in the younger age group, with that group's follow-on project, Prevnar 20, due to yield phase 3 in the second half of the year. Lastly Bristol Myers Squibb finally received the green light for mavacamten, now called Camzyos, in obstructive hypertrophic cardiomyopathy. The cardiac myosin inhibitor not only comes with a REMS and a black boxed warning, but a higher than expected price tag. With a list price of $89,500 a year it is well above the $15,000 figure ICER said would meet traditional thresholds of cost-effectiveness. Bristol has its work cut out to meet its $4bn-plus peak sales target, and justifying the $13.1bn it spent on Myokardia.

Notable first-time US approval decisions in April
Project Company Indication(s) 2026e sales by indication (SBI) ($m) Outcome
Vutrisiran Alnylam Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis 1,828* Delayed to July 14 (review of new information related to the secondary packaging and labelling facility)
Camzyos (mavacamten) Bristol Myers Squibb Obstructive hypertrophic cardiomyopathy 1,672 Approved 
Bimzelx UCB Plaque psoriasis 953 Pending (H1)
Igalmi
(dexmedetomidine/ BXCL501)
Bioxcel Agitation associated with schizophrenia and bipolar disorders I and II 378 Approved 
Sulanda
(surufatinib)
Hutchmed Pancreatic and non-pancreatic neuroendocrine tumors 362 CRL (multi-regional clinical trial needed)
FT218 Avadel Narcolepsy 321 Pending
AXS-07 Axsome Acute treatment of migraine 128 CRL (CMC issues)
Epsolay cream Sol-Gel/ Galderma Papulopustular rosacea - Approved 
Vivjoa
(oteseconazole)
Mycovia Recurrent vulvovaginal candidiasis - Approved 
Cuvrior (Cuprior) Orphalan Adult patients with stable Wilson’s disease - Approved 
Tuoyi (toripalimab) Coherus/ Shanghai Junshi 1st-line chemo combo & 3rd-line monoRx nasopharyngeal carcinoma - CRL (quality process change required)
Tapinarof Dermavant Plaque psoriasis - Pending (Q2)
Annik
(penpulimab)
Akeso/Sino 3L nasopharyngeal carcinoma - Pending (H1)
SH-111 Shorla Oncology (private) T-cell leukaemia - Pending
*SBI as amyloidosis. Source: company statements, Evaluate Pharma

 

Advisory committee meetings in April
Project Company Indication Outcome
Covid-19 vaccination N/A Covid-19 prevention Agency will reconvene its booster discussion on June 28
PI3K inhibitor class N/A Haematologic malignancies  16-0, future approvals of PI3K inhibitors should be supported by randomized data
U2, Ukoniq TG Therapeutics Haematologic malignancies  Prior to ad com U2 application withdrawn and Ukoniq pulled
Source: FDA ad com calendar, Evaluate Pharma

 

Supplementary and other notable approval decisions in April
Product Company Indication (clinical trial) Outcome
Vaxneuvance Merck Prevention of invasive pneumococcal disease in children 6 weeks through 17 years of age (15-valent conjugate vaccine, PNEU-PED) Delayed to July 1 (additional analyses of data from the paediatric studies)
REgen-Cov
(Ronapreve)
Regeneron Treat Covid-19 in non-hospitalised patients and as prophylaxis in certain individuals Delayed to July 13 (already has EUA)
Yescarta Gilead 2L relapsed/refractory large B-cell lymphoma (Zuma-7) Approved
Qelbree Supernus ADHD in adults (P306) Approved 
Ultomiris Astrazeneca Adults with generalised myasthenia gravis who are anti-acetylcholine receptor antibody-positive (NCT03920293) Approved 
Rinvoq Abbvie Ankylosing spondylitis (Select-Axis 2, Select-Axis 1) Approved
Vijoice (alpelisib, marketed as Piqray in breast cancer) Novartis Adult and paediatric patients 2 years of age and older with PIK3CA-related overgrowth spectrum (EPIK-P1) Approved 
Cysteine hydrochloride (Elcys generic) Eton Additive to amino acid solutions to meet the nutritional requirements of newborn infants Approved 
Alymsys (Avastin biosimilar) Amneal/ mAbxience Metastatic colorectal cancer, 1L NSCLC, recurrent glioblastoma, met renal cancer, met cervical cancer, epithelial ovarian, fallopian tube, or primary peritoneal cancer Approved 
Veklury Gilead Paediatric patients under 12 years old hospitalised with Covid-19 or have mild-to-moderate Covid-19 at high risk of progression to severe Covid-19 Approved 
Caplyta Intra-cellular New dosage strengths for schizophrenia and bipolar depression Approved 
Olumiant Lilly Atopic dermatitis (Breeze-AD programme) CRL
TV-46000/mdc-IRM (risperidone extended-release) Teva/ MedinCell Schizophrenia (Rise, Shine) CRL
Enhertu Astra/Daiichi Unresectable/metastatic Her2 +ve breast cancer pts who have received a prior anti-Her2-based regimen (Destiny-Breast 03) Pending (Q2)
Olumiant Lilly Treatment of certain hospitalised patients with Covid-19 Pending (Q2)
Kymriah Novartis r/r FL after two prior lines of treatment (Ph2 Elara) Pending
Source: company statements, Evaluate Pharma

 

FDA Covid-19 EUAs
Product Company Outcome
Xevudy
(sotrovimab)
GSK/Vir No longer authorised to treat Covid-19 in any US region as Xevudy is unlikely to be effective against the BA.2 sub-variant (gained EUA in May 2021)
Source: company releases

Share This Article

 
Vantage logo
Independent, data-driven daily news and analysis on pharma, biotech and medtech.