US FDA approval tracker: August 2022
It was a double FDA thumbs-up for Astrazeneca and Daiichi Sankyo’s Enhertu last month as the antibody-drug conjugate became the first Her2-targeted agent approved in Her2-positive lung cancer and Her2-low breast cancer. Enhertu looks set to dominate the Her2-low space, a newly defined subset in breast cancer, as competitors are still some way behind. Bluebird also received approval for Zynteglo, a gene therapy for transfusion-dependent beta-thalassaemia, but the company has a huge commercial task ahead to try and make the product profitable. Bluebird’s second gene therapy, eli-cel, has a Pdufa date in September, this time for cerebral adrenoleukodystrophy, a rare metabolic disorder. Investors will have to wait a little longer for several pending FDA decisions, including J&J’s multiple myeloma bispecific Tecvayli, which gained European approval in fourth-line disease last month.
| Notable first-time US approval decisions in August | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e sales by indication (SBI) ($m) | Outcome |
| Auvelity (AXS-05) |
Axsome | Major depressive disorder | 787 | Approved |
| Neutrolin/ Defencath |
Cormedix | Prevention of catheter-related bloodstream infections | 392 | CRL (second, this time due to deficiencies at CMO) |
| Xenpozyme | Sanofi | Non-CNS manifestations of acid sphingomyelinase deficiency (Niemann-Pick disease) in adult and paediatric patients | 346 | Approved (~1 month early) |
| Miglustat for AT-GAA | Amicus | Pompe disease | 266* | No decision yet (part of two-component therapy with cipaglucosidase, Pdufa in Oct) |
| Tecvayli (teclistamab) | Johnson & Johnson | R/R multiple myeloma | 260 | No decision yet |
| Betibeglogene autotemcel (Zynteglo/ beti-cel) | Bluebird | Beta-thalassaemia (pts requiring regular red blood cell transfusions) | 120 | Approved |
| Libervant (diazepam) buccal film | Aquestive | Acute treatment of intermittent, stereotypic episodes of frequent seizure activity | 90 | Tentative approval (Neurelis' Valtoco has orphan drug exclusivity) |
| Annik (penpulimab) | Akeso/Sino | 3L nasopharyngeal carcinoma | - | No decision yet |
| *Forecast for AT-GAA. Source: company releases & Evaluate Pharma. | ||||
| Private companies with pending FDA approvals | |||
|---|---|---|---|
| Project | Company | Indication | Outcome |
| SH-111 | Shorla Oncology | Undisclosed project for T-cell leukaemia | Pending (was filed in Apr 2021) |
| Spesolimab | Boehringer Ingelheim | Generalised pustular psoriasis | Decision expected by YE |
| Source: company releases & Evaluate Pharma. | |||
| Supplementary and other notable approval decisions in August | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Nuplazid | Acadia | Hallucinations and delusions associated with Alzheimer's disease psychosis (Harmony, phase 2) | CRL (new study required) |
| Myfembree | Pfizer/ Myovant |
Moderate-to-severe pain associated with endometriosis (Spirit 1, Spirit 2) | Approved |
| Enhertu | Astra/Daiichi Sankyo | 2L Her2-positive NSCLC (Destiny-Lung01, Destiny-Lung02) | Approved (accelerated) |
| Unresectable metastatic Her2 low breast cancer (Destiny-Breast04) | Approved (~2 months early) | ||
| Cimerli (CHS-201/ FYB201, Lucentis biosimilar) | Teva/Bioeq/ Formycon/ Coherus |
Wet AMD, macular oedema following retinal vein occlusion, DME, diabetic retinopathy, and myopic choroidal neovascularisation | Approved |
| Haloette (generic Nuvaring) | Mayne Pharma/ Mithra |
Vaginal hormonal contraceptive ring | Approved (three previous CRLs) |
| Nubeqa + docetaxel | Bayer/ Orion |
Metastatic hormone-sensitive prostate cancer (Arasens) | Approved |
| Calquence tablet | Astrazeneca | Adult patients with chronic lymphocytic leukaemia, small lymphocytic lymphoma and for patients with relapsed or refractory mantle cell lymphoma (Elevate-Plus) | Approved |
| Xofluza | Roche | Treatment of influenza in children aged 5-12, and for the prevention of influenza following contact with someone with influenza (oral therapy) (Ministone-2, Blockstone) | Approved |
| Hadlima (High concentration Humira biosimilar) (SB5) | Samsung Bioepis/Organon | RA, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn's, ulcerative colitis, plaque psoriasis (NCT04514796) | Approved (launch expected on/after July 1 2023) |
| Tabrecta | Novartis | Adult patients with metastatic NSCLC whose tumours have a mutation leading to MET exon 14 skipping (Geometry mono-1) | Full approval (accelerated approval granted 2020) |
| Pemazyre | Incyte | Myeloid/lymphoid neoplasms with FGFR1 rearrangement (Ph2 Fight-203) | Approved |
| Imbruvica | J&J | Paediatric patients one year and older with chronic graft-versus-host disease after failure of one or more lines of systemic therapy (Ph1/2 Imagine) | Approved |
| Source: company releases & Evaluate Pharma. | |||
| FDA EUAs | ||
|---|---|---|
| Product | Company | Indication/outcome |
| Jynneos | Bavarian Nordic | Approved for use in individuals determined to be at high risk for monkeypox infection |
| Nuvaxovid | Novavax | Prevention in Covid-19 in individuals aged 12-17 years old (vaccine, first granted an EUA in July) |
| Bivalent Covid-19 vaccine (mRNA-1273.222) | Moderna | For use as a booster dose, includes original Sars-Cov-2 strain and omicron variant |
| Bivalent Covid-19 vaccine | Pfizer/Biontech | |
| Source: FDA, company releases & Evaluate Pharma. | ||
