US approval tracker: January 2023
Last month was a tale of two Alzheimer's MAbs, with celebrations for Eisai and commiserations for Lilly. Eisai’s lecanemab, now branded Leqembi, gained an accelerated approval in Alzheimer’s patients with mild cognitive impairment or mild dementia. A day after the greenlight Eisai filed for full approval. Lilly’s donanemab, on the other hand, was knocked back, as the filing did not include enough patients with at least 12 months' exposure to the MAb. Donanemab’s confirmatory phase 3 data are due mid-year. In oncology there were a couple of approval firsts in Menarini’s oral Serd Orserdu and Lilly’s non-covalent BTK inhibitor Jaypirca. Also in oncology, Beigene’s covalent BTK inhibitor Brukinsa added chronic lymphocytic leukaemia to its label. The label includes a superiority claim over Johnson & Johnson/Abbvie’s Imbruvica on overall response rate, and Beigene is planning to submit a post-approval labelling supplement to get PFS superiority added. The latter could help Brukinsa take market share from Astrazeneca’s Calquence, which has only demonstrated non-inferiority to Imbruvica.
| Notable first-time US approval decisions in January | ||||
|---|---|---|---|---|
| Project | Company | Indication(s) | 2028e SBI ($m) | Outcome |
| Donanemab | Lilly | Early symptomatic Alzheimer's disease | 1,872* | CRL |
| Leqembi (lecanemab) | Eisai/Biogen | Alzheimer's disease | 1,854 | Approved (accelerated) |
| Orserdu (elacestrant) | Menarini (private)/ Radius |
2L and 3L ER+/Her2- advanced/metastatic breast cancer | - | Approved (~2 weeks early) |
| Jaypirca (pirtobrutinib) | Lilly/Loxo | Mantle cell lymphoma for patients previously treated with a BTK inhibitor | -** | Approved |
| Airsupra (PT027) | Astrazeneca/ Avillion | As-needed treatment or prevention of bronchoconstriction and to reduce the risk of exacerbations in asthma pts aged ≥18 | - | Approved in adults; CRL in children aged 4-17 (negative adcom in November) |
| Brenzavvy (bexagliflozin) | Theracosbio | Adjunct to diet and exercise to improve glycaemic control in adults with type 2 diabetes | - | Approved |
| *Forecast prior to CRL, **sales assigned to CLL. SBI: sales by indication. Source: Evaluate Pharma, company releases. | ||||
| Advisory committee meetings in January | ||||
|---|---|---|---|---|
| Project | Company | Indication | 2028e SBI ($m) | Outcome |
| Rezafungin | Cidara/ Melinta/ Mundipharma |
Candidemia and invasive candidiasis in adults | 348 | 14-1 in favour, in adults with limited or no alternative treatment options (Pdufa Mar 22) |
| Eylea | Regeneron | Retinopathy of prematurity in preterm infants | - | No vote; discussions were around appropriate communication and labelling. No anti-VEGF treatments have been approved in the US for the indication but several are used off-label (Pdufa Feb 11) |
| - | - | Future Covid-19 vaccination regimens | - | Recommended that all future vaccines should be bivalent. Another meeting will take place in May/June to discuss the latest variant data and whether the current booster shots will be sufficient for the autumn |
| Source: Evaluate Pharma, company releases, FDA adcom calendar. | ||||
| Supplementary and other notable approval decisions in January | |||
|---|---|---|---|
| Product | Company | Indication (clinical trial) | Outcome |
| Vonaprazan (Takecab) | Phathom | Erosive oesophagitis (Phalcon-ee) | Delayed (impurity found in commercial batches, additional stability data needed. Also affects approved H pylori indication) |
| Tukysa + Herceptin | Seagen | Adult patients with Her2-positive colorectal cancer who have received at least 1 prior treatment regimen for unresectable or metastatic disease (Ph2 Mountaineer) | Approved (accelerated) |
| Brukinsa | Beigene | Adults with CLL or SLL (Alpine, Sequoia) | Approved |
| Keytruda | Merck & Co | Adjuvant therapy for Stage IB (≥4 cm)-IIIA NSCLC following complete resection (Pearls/Keynote-091) | Approved |
| Myfembree | Pfizer/Myovant | Update to prescribing information (heavy menstrual bleeding associated with uterine fibroids) based on safety and efficacy data from the Liberty randomised withdrawal study | Approved |
| Rybelsus | Novo Nordisk | 1L treatment option for adults with type 2 diabetes | Approved |
| Rykindo (risperidone for extended-release injectable suspension) | Luye Pharma | Schizophrenia in adults and as monotherapy or as adjunctive therapy to lithium or valproate for the maintenance treatment of bipolar I disorder in adults | Approved |
| Vabysmo | Roche | Include two-year data from Yosemite and Rhine studies in diabetic macular oedema | Approved |
| Evusheld | Astrazeneca | Pre-exposure prophylaxis of Covid-19 | EUA removed due to lack of efficacy against circulating variants |
| Acalabrutinib (generic to Astrazeneca's Calquence) | Alembic Pharms | Mantle cell lymphoma (after at least one prior therapy) and chronic lymphocytic leukaemia or small lymphocytic lymphoma | Tentative approval |
| Insugen (rh-insulin) (biosimilar) | Biocon | Diabetes | CRL (additional data, inspection issues) |
| Source: Evaluate Pharma, company releases. | |||
