
US FDA approval tracker: April 2022
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 $13.1bn it spent on Myokardia.
Notable first-time US approval decisions in April | ||||
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Project | Company | Indication(s) | 2026e sales by indication (SBI) ($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 |