The FDA dished out three Pdufa extensions and four knockbacks in November, including a CRL for Beyondspring’s plinabulin in chemotherapy-induced neutropenia. Beyondspring said today that the FDA had requested a second clinical study of plinabulin; this news sent the company's shares down 55% in early trading and follows disappointing NSCLC data at Esmo. On the flip side, Biomarin’s Voxzogo gained accelerated approval in patients aged five and older with achondroplasia. Assessing final adult height is part of the post-marketing requirement and Biomarin has said it will use the ongoing open-label extension studies versus natural history for confirmatory data. Merck’s Keytruda also gained another string to its bow last month with an adjuvant approval in renal cell cancer. The decision came three weeks early and puts Keytruda further ahead of rivals, which will start reporting phase 3 data next year.
| Notable first-time US approval decisions in November | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2026e SBI ($m) | Outcome |
| Ciltacabtagene autoleucel (cilta-cel) | Johnson & Johnson/ Legend Biotech | 3L+ multiple myeloma | 1,692 | Extended to Feb 28 (updated analytical method) |
| Voxzogo (vosoritide) | Biomarin | Achondroplasia | 720 | Approved (accelerated) |
| Doria (risperidone ISM) | Laboratorios Farmacéuticos ROVI | Schizophrenia | 375 | CRL (manufacturing inspection) |
| Enpaxiq (pacritinib) | CTI Biopharma | Myelofibrosis patients with severe thrombocytopenia | 368 | Extended to Feb 28 (data submission) |
| Livtencity (maribavir) | Takeda | Post-transplant cytomegalovirus infection in those that are refractory, with or without resistance | 355 | Approved |
| Oleogel-S10 (Filsuvez) | Amryt | Epidermolysis bullosa | 269 | Extended to Feb 28 (review of additional data analyses) |
| Erwinaze | Clinigen/ Porton Biopharma | ALL patients who have developed hypersensitivity to E. coli-derived asparaginase | 211 | CRL |
| Eohilia (TAK-721) | Takeda | Eosinophilic oesophagitis | 188 | No decision yet |
| Pedmark | Fennec | Cisplatin-induced ototoxicity for paediatric patients | 165 | CRL (manufacturing deficiencies) |
| PreHevbrio (Sci-B-Vac) | Opko Health/ VBI Vaccines | Hepatitis B | 164 | Approved |
| Plinabulin + G-CSF | Beyondspring | Prevention of chemotherapy-induced neutropenia | 138 | CRL (more data requested) |
| Yutrepia (LIQ861 /treprostinil inhalation powder) | Liquidia | Pulmonary arterial hypertension | 126 | Tentative approval* |
| Cytalux (OTL38/ pafolacianine sodium injection) | On Target Laboratories | Adjunct for identifying ovarian cancer during surgery | - | Approved |
| Eprontia (topiramate oral solution /ET-101) | Azurity Pharmaceuticals/ Eton Pharmaceuticals | Tonic-clonic seizures, partial-onset seizures, and as preventative treatment of migraine | - | Approved |
| Besremi (ropeginterferon alfa-2b) | Pharmaessentia | Polycythemia vera | - | Approved |
| Fyarro (ABI-009) | Aadi Bioscience | Advanced malignant PEComa | - | Approved |
| Epsolay | Sol-Gel | Papulopustular rosacea | - | No decision yet |
| *Final FDA approval may occur in Oct 2022 or earlier upon resolution of ongoing litigation with United Therapeutics. SBI=sales by indication, CMC=chemistry, manufacturing and controls. | ||||
| Source: Evaluate Pharma & company releases. | ||||
| Advisory committee meetings in November | |||
|---|---|---|---|
| Project | Company | Indication | Outcome |
| LV-101 (intranasal carbetocin) | Levo | Hyperphagia, anxiety, and distress behaviours associated with Prader-Willi syndrome | 12-1 against approval |
| Molnupiravir | Merck/ Ridgeback | EUA for the treatment of mild to moderate Covid-19 in adults who are at risk for progressing to severe Covid-19 and/or hospitalisation | 13-10 for EUA |
| Source: FDA adcom calendar, Evaluate Pharma. | |||
| Supplementary and other notable approval decisions in November | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Dyanavel XR tablets | Tris Pharma | ADHD | Approved |
| Keytruda | Merck | Adjuvant treatment of RCC at intermediate-high or high risk of recurrence (Keynote-564) | Approved (3 weeks early) |
| Pobevcy (BAT1706) (Avastin biosimmilar) | Bio-Thera Solutions | Metastatic colorectal cancer (+ chemo), 1L non-squamous NSCLC, recurrent glioblastoma, met renal cell carcinoma (+ interferon alfa), persistent, recurrent or met cervical cancer | No decision yet |
| Source: Evaluate Pharma & company releases. https://www.evaluate.com/vantage/articles/events/company-events-snippets/us-fda-approval-tracker-november | |||
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