Global News
Pfizer announced the voluntary withdrawal of all lots of Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) from all markets where it is currently approved. Pfizer is also discontinuing all active Oxbryta trials and expanded access programs globally. This decision is based on data that revealed an imbalance in fatalities and complications commonly associated with the disease, according to a Pfizer press release. Pfizer press release.
Sage Therapeutics revealed that Biogen has terminated its rights under the collaboration and license agreement for the SAGE-324 program following negative results from its Phase 2 study for the chronic treatment of essential tremor (ET). Sage press release.
European Medicine Agency
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) held its monthly meeting from September 30 to October 3. Key takeaways include:
- Initiation of a review of medicines containing finasteride and dutasteride due to concerns about suicidal ideation (suicidal thoughts) and behaviors.
- Direct Healthcare Professional Communication (DHPC) regarding a change to the dosing syringe included in the product packaging of Keppra and Levetiracetam UCB 100 mg/ml oral solution, intended for use in children aged 6 months to 4 years old (150 ml bottle). The DHPC will inform healthcare professionals of the potential risk of medication errors stemming from the change in the volume of the dosing syringe. PRAC meeting highlights.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) will hold its monthly meeting from October 14-17, 2024.
US Food and Drug Administration (FDA)
The FDA did take the following decisions in the last 3 weeks:
Dasiglucagon (Zealand Pharma) was rejected by the FDA for the prevention and treatment of hypoglycemia in pediatric patients aged 7 days and older with congenital hyperinsulinism (CHI). The Complete Response Letter (CRL) cited issues with the timing of the re-inspection of a third-party manufacturing facility. Zealand Pharma press release.
Bimzelx (bimekizumab-bkzx, UCB) was approved for treating adults with active psoriatic arthritis (PsA), active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation, and active ankylosing spondylitis (AS). UCB press release.
Retevmo (selpercatinib, Eli Lilly) was approved for adults and pediatric patients aged 2 years and older with advanced or metastatic medullary thyroid cancer (MTC) harboring a RET mutation. FDA press release.
Cobenfy (xanomeline and trospium chloride, BMS) was approved for the treatment of schizophrenia in adults. BMS press release.
Opdivo (nivolumab, BMS) received label expansion approval as a neoadjuvant treatment for adults with resectable non-small cell lung cancer (NSCLC). BMS press release.
Aqneursa (levacetylleucine, IntraBio) was approved for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥ 15 kg. IntraBio press release.
Otulfi (ustekinumab-aauz, Fresenius Kabi and Formycon) was approved as a biosimilar to Stelara (ustekinumab, Johnson & Johnson) for the treatment of Crohn’s disease, ulcerative colitis, moderate to severe plaque psoriasis and active psoriatic arthritis. Fresenius Kabi press release.
Tagrisso (Osimertinib, AstraZeneca) received label expansion approval for the treatment of adult patients with unresectable Stage III epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC). AstraZeneca press release.
Dupixent (dupilumab, Sanofi and Regeneron) was approved as an add-on maintenance treatment for adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic phenotype. Sanofi press release.
Hympavzi (marstacimab-hncq, Pfizer) was approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients aged 12 years and older with hemophilia A or B without inhibitors. Pfizer press release.
Itovebi (inavolisib, Roche) was approved for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer. Roche press release.
New data from clinical trials (Phase 2, Phase 3)
Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between the September 23rd and October 13th,2024:
InnoCare Pharma announced that the Phase 2 study results of ICP-488 in adult patients with moderate-to-severe plaque psoriasis met its primary endpoint. InnoCare press release.
Rivus Pharmaceuticals shared positive data from the Phase 2a trial of HU6 in patients with obesity-related heart failure with preserved ejection fraction (HFpEF). Rivus press release.
Remedy Pharmaceuticals reported positive data from the Phase 3 trial of CIRARA (IV glyburide) in patients with Large Hemispheric Infarction (LHI). Remedy press release.
Roche announced that the Phase 3 study of Gazyva/Gazyvaro (obinutuzumab) in patients with active lupus nephritis met its primary endpoint. Roche press release.
UCB and Biogen shared positive results from the Phase 3 study of dapirolizumab pegol in patients with moderate-to-severe systemic lupus erythematosus (SLE). Biogen press release.
Biohaven announced that the pivotal study of Troriluzole in patients with Spinocerebellar Ataxia (SCA) met its primary endpoint. Biohaven press release.
Wave Life Sciences reported positive data from the Phase 2 study of WVE-N531 in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. Wave press release
AbbVie shared positive results from the Phase 3 trial of tavapadon as a monotherapy in early Parkinson’s disease. AbbVie press release.
Travere Therapeutics announced a voluntary enrollment pause in its Phase 3 trial of pegtibatinase for treating classical homocystinuria (HCU) to address manufacturing scale-up issues. Travere press release.
2seventy bio (and BMS) announced the discontinuation of the Phase 3 study evaluating Abecma (idecabtagene vicleucel; ide-cel) with lenalidomide in patients with newly diagnosed multiple myeloma (NDMM). 2seventy bio press release.
Galderma presented additional long-term data from two Phase 3 trials of nemolizumab in atopic dermatitis and prurigo nodularis. Galderma press release. The data were presented at EADV 2024 congress.
IDEAYA Biosciences shared positive Phase 2 data for darovasertib in neoadjuvant uveal melanoma (UM). IDEAYA Biosciences press release.
AbbVie reported positive data from two Phase 3 trials of Rinvoq (upadacitinib) in patients with moderate-to-severe atopic dermatitis (AD). AbbVie press release.
Union Therapeutics shared positive results from the Phase 2b study of orismilast in adults with moderate to severe atopic dermatitis. Union Therapeutics press release.
Kezar Life Sciences announced a voluntary halt on new patient enrollment and ongoing dosing in its Phase 2b trial of zetomipzomib for active lupus nephritis. This decision follows recommendations from an independent data monitoring committee after reviewing safety data from the trial. According to the safety findings, four Grade Five (fatal) serious adverse events were reported during the trial. Kezar Life Sciences press release.
Biomea Fusion announced that the FDA lifted the clinical hold on its ongoing Phase 1/2 trials of BMF-219 in type 2 and type 1 diabetes. Biomea press release.
Johnson & Johnson announced the discontinuation of its Phase 2 study evaluating mosnodenvir for the prevention of dengue virus in adults aged 18-65. This decision is part of the company’s strategic reprioritization of its research and development (R&D) efforts in communicable diseases. J&J press release.
Sage Therapeutics also announced that the Phase 2 study of dalzanemdor (SAGE-718) in participants with mild cognitive impairment (MCI) or mild dementia due to Alzheimer’s Disease (AD) did not meet its primary endpoint. Based on these results, Sage will discontinue further development of dalzanemdor in AD. Sage press release.
GSK presented positive results from a Phase 3 trial evaluating a single dose of Arexvy (recombinant adjuvanted RSV vaccine) for its efficacy against lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in adults aged 60 and older. GSK press release.
Scholar Rock reported that its Phase 3 trial of apitegromab in patients with spinal muscular atrophy (SMA) successfully met its primary endpoint. Scholar Rock press release.
Pfizer shared positive results from the Phase 3 study of Talzenna (talazoparib) in combination with Xtandi (enzalutamide) in patients with metastatic castration-resistant prostate cancer (mCRPC). Pfizer press release.
Johnson & Johnson announced the termination of its Phase 3 study of TAR-200 for muscle-invasive bladder cancer (MIBC) in patients not undergoing radical cystectomy. The decision followed a recommendation from an independent data monitoring committee after a pre-specified interim analysis. J&J press release.
AstraZeneca reported that its Phase 3b trial of Airsupra (albuterol/budesonide) in patients with intermittent or mild persistent asthma met its primary endpoint. AstraZeneca press release.
Merck announced that the Phase 3 trial of Keytruda (pembrolizumab) for the treatment of head and neck squamous cell carcinoma (HNSCC) met its primary endpoint. Merck press release.
Cited Articles
Labade et al 2024: Ajay S. Labade, Zachary D. Chiang, Caroline Comenho, Paul L. Reginato, Andrew C.Payne, Andrew S. Earl, Rojesh Shrestha, Fabiana M. Duarte, Ehsan Habibi, Ruochi Zhang, George M. Church, Edward S. Boyden, Fei Chen, Jason D. Buenrostro. bioRxiv 2024.09.24.614614; doi: https://doi.org/10.1101/2024.09.24.614614
Pannone et al 2024: Pannone, A., Raj, A., Ravichandran, H. et al. Robust chemical analysis with graphene chemosensors and machine learning. Nature (2024). https://doi.org/10.1038/s41586-024-08003-w
