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 GSK’s Zejula, on the other hand, was voluntarily withdrawn for late-line use in ovarian cancer owing to a potential survival detriment with the Parp inhibitor class.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
Amylyx ALS 1,362 Approved (full approval)
Daxxify (DaxibotulinumtoxinA, Daxi) Revance Moderate-to-severe glabellar lines 510 Approved (prior CRL)
(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)
Boehringer Ingelheim Generalised pustular psoriasis flares - Approved
Iheezo (chloroprocaine hydrochloride ophthalmic gel 3%, AMP-100) Harrow Health/
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
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/
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.

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