US FDA approval tracker: September 2022
A green light was finally bestowed on Amylyx’s ALS therapy last month after a positive second FDA panel. Relyvrio, as AMX0035 is now called in the US, gained full approval, but a risk remains: failure of the pivotal Phoenix study, data from which are due in 2024, might result in the product getting pulled from the market. Amylyx had pledged to price Relyvrio below the most recently approved ALS therapy, which SVB analysts noted was Mitsubishi Tanabe’s Radicava at $165,000 per year. However, Relyvrio's $158,000 per year was only just below this, and much higher than Icer’s recommended $9,100-30,700 annually. Another high-profile FDA approval was that of Bristol Myers Squibb’s Tyk2 inhibitor Sotyktu in psoriasis, with a better than expected label. Elsewhere Revance, Spectrum, Fennec and Mallinckrodt all received green lights after previous complete response letters. GSK’s Zejula, on the other hand, was voluntarily withdrawn for late-line use in ovarian cancer owing to a potential survival detriment with the Parp inhibitor class. An adcom in November will discuss Zejula’s ongoing use.
| Notable first-time US approval decisions in September | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e sales by indication (SBI) ($m) | Outcome |
| Sotyktu (deucravacitinib) | Bristol Myers Squibb | Moderate-to-severe psoriasis | 1,633 | Approved |
| Relyvrio/Albrioza (AMX0035) |
Amylyx | ALS | 1,362 | Approved (full approval) |
| Daxxify (DaxibotulinumtoxinA, Daxi) | Revance | Moderate-to-severe glabellar lines | 510 | Approved (prior CRL) |
| Rolvedon (Rolontis, eflapegrastim) |
Spectrum | Chemotherapy-induced neutropenia | 297 | Approved (prior CRL) |
| Tecvayli (teclistamab) | Johnson & Johnson | r/r multiple myeloma | 260 | Pending |
| Omlonti | Santen/Ube | Reducing elevated intraocular pressure in patients with open‑angle glaucoma or ocular hypertension | 145 | Approved |
| Pedmark | Fennec | Prevention of platinum-induced ototoxicity in paediatric patients | 137 | Approved (prior CRL) |
| Terlivaz (terllipressin) | Mallinckrodt | Hepatorenal syndrome | 118 | Approved (two previous CRLs) |
|
Lytgobi (futibatinib, TAS-120) |
Taiho Oncology/Otsuka | Patients with previously treated locally advanced or metastatic cholangiocarcinoma harbouring FGFR2 gene rearrangements, including gene fusions | 80 | Approved (accelerated) |
| Elivaldogene autotemcel (eli-cel, Skysona) | Bluebird | Cerebral adrenoleukodystrophy | 21 | Approved (accelerated) |
| Spevigo (spesolimab) |
Boehringer Ingelheim | Generalised pustular psoriasis flares | - | Approved |
| Iheezo (chloroprocaine hydrochloride ophthalmic gel 3%, AMP-100) | Harrow Health/ Sintetica |
Ocular surface anaesthesia and intraoperative pain management | - | Approved |
| Elucirem (gadopiclenol) | Guerbet | Detect and visualise lesions, together with MRI, with abnormal vascularity in the CNS and the body | - | Approved |
| Annik (penpulimab) | Akeso/Sino | 3L nasopharyngeal carcinoma | - | Pending |
| Source: Evaluate Pharma & company releases. | ||||
| Advisory committee meetings in September | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Relyvrio/Albrioza (AMX0035) |
Amylyx | ALS | 1,362 | 7-2 in favour (Amylyx’s fortunes shift again) |
| Poziotinib | Spectrum | Previously treated Her2 exon 20 insertion mutatated NSCLC | 346 | 9-4 against (Zentih20 study) |
| Pepaxto (melphalan flufenamide) | Oncopeptides | R/r multiple myeloma after at least 4 lines of therapy, disease refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed MAb | 141 | 14-2 against (Ocean study) |
| Rebyota (RBX2660) | Ferring (private) (via Rebiotix) | Reduce the recurrence of Clostridioides difficile infection in adults following antibiotic treatment for recurrent C diff infection | - | 13-4 in favour of effectiveness, 12-4 with 1 abstention in favour of safety (Ferring gets a yes, but a Seres threat is looming) |
| Copiktra (duvelisib) | Secura Bio/Verastem/ Infinity |
Adult patients with relapsed or refractory chronic lymphocytic leukaemia or small lymphocytic lymphoma after at least two prior therapies | - | 8-4 against (Duo study) |
| Source: Evaluate Pharma, company releases & FDA adcom calendar. | ||||
| Supplementary and other notable approval decisions in September | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Orkambi | Vertex | Patients with cystic fibrosis aged 12mth to <24mth | Approved |
| Dupixent | Sanofi/Regeneron | Prurigo nodularis (Prime 2 and Prime) | Approved |
| Imfinzi + chemo | Astrazeneca | 1L biliary tract cancer (Topaz-1) | Approved |
| Stimufend (Neulasta biosimilar) | Fresenius | Decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anticancer drugs | Approved |
| Xtandi | Astellas | Metastatic hormone-sensitive prostate cancer (include OS data from Arches study) | Approved |
| Aponvie (Cinvanti/ HTX-019) | Heron | Injectable emulsion, for intravenous use for preventing postoperative nausea and vomiting in adults (30-sec injection) | Approved |
| Tabreta | Novartis/Incyte | NSCLC with Met exon 14 skipping mutation, as detected by FDA-approved test (Geometry mono-1) | Regular approval (had been accelerated) |
| Retevmo | Lilly | NSCLC with a Ret gene fusion (Libretto-001) | Regular approval (had been accelerated) |
| Retevmo | Lilly | Solid tumours with Ret gene fusion, tumour-agnostic (Libretto-001) | Accelerated approval |
| Vegzelma (Avastin biosimilar) | Celltrion | mCRC, recurrent or metastatic nsNSCLC, recurrent GBM, mRCC, persistent, recurrent, or metastatic CC, epithelial ovarian, fallopian tube, or primary peritoneal cancer | Approved |
| Rifaximin 200mg (Xifaxan generic) | Norwich Pharmaceuticals | Travellers' diarrhoea | Tentative approval (full approval not expected until expiry of last patent in 2029) |
| Zejula | GSK | Ovarian cancer treated with three or more prior chemotherapy regimens (Quandra) | Voluntarily withdrawn, 22 Nov adcom to discuss OS data from Nova study in patients in response to platinum chemo |
| AVT02 (Humira biosimilar, high concentration) |
Alvotech/Teva | Inflammatory conditions | CRL (manufacturing deficiencies) |
| Libtayo | Regeneron | 1L NSCLC (+chemo; Empower-Lung 3) | Pending (delay had been expected owing to clinical trial site inspection in Eastern Europe) |
| mCRC; metastatic colorectal cancer, nsNSCLC; non-squamous non-small cell lung cancer, GBM; glioblastoma, mRCC; metastatic renal cell carcinoma, CC; cervical cancer. Source: Evaluate Pharma & company releases. | |||
