Drugs
A Federation of American Scientists online report calls on FDA to create a dedicated regulatory pathway for non-manufacturers to seek approval of new indications for repurposed generic drugs. Written by three RebootRx executives, the report says one possible approach would be an extension of the existing 510(b)(2)
Drugs
FDA has accepted for review a Verastem Oncology NDA for avutometinib, an oral RAF/MEK clamp for use in combination with defactinib, an oral FAK inhibitor, for treating adult patients with recurrent low-grade serous ovarian cancer (LGSOC) who received at least one prior systemic therapy and have a KRAS
Biologics
An FDA summary report on clinical study diversity action plans for FY 2023 and FY 2024 shows that submission of the action plans to the agency is going up, even though such submissions are not yet required. The report shows that CDER received 124 plans in FY 2023 and 161
Drugs
A 1/3-1/5/2024 FDA inspection at XO Biologix in Austin, TX, determined that the company markets and distributes a product derived from amniotic fluid under a redacted contract. A just-released 12/12/2024 Warning Letter says the company’s MaviX product is an unapproved new drug
Drugs
FDA has granted fast-track designation for Johnson & Johnson’s posdinemab which is being investigated in the Phase 2b AuTonomy study to treat patients with early Alzheimer’s disease. A company statement says posdinemab is a phosphorylate tau-directed monoclonal antibody that was discovered internally and has shown the
Drugs
First-cycle NDA approvals fell in 2024 to 74% (37 of the 50 novel drugs approved) from 84% (46 of the 55 novel approvals) in 2023, according to CDER’s annual new drug approvals report. In contrast, CDER approved 28 of the 37 (76%) novel drugs on the first cycle
Drugs
The FDA Anesthetic and Analgesic Drug Products Advisory Committee will discuss 1/10 the challenges FDA has identified in a Seikagaku BLA for condoliase with a proposed indication to treat radicular leg pain associated with confirmed nerve root impingement caused by lumbar disc herniation in adults. An agency briefing document
Drugs
Vanda Pharmaceuticals is criticizing FDA for failing to approve the company’s NDA for tradipitant to treat the symptoms of gastroparesis. In 9/2024, FDA issued a complete response letter that the company said was “conclusory in nature, generally disregarded the evidence provided and instead suggested that Vanda conduct additional
Drugs
FDA has published a draft guidance, Developing Drugs for Optical Imaging, with recommendations for sponsors about clinical trial design features that support the development and approval of optical imaging drugs that are used in conjunction with imaging devices and intended as intraoperative aids for the detection of pathology such as
Drugs
FDA has accepted for priority review biopharmaceutical firm Dizal’s NDA for sunvozertinib, an oral EGFR inhibitor for treating locally advanced or metastatic non-small cell lung cancer (NSCLC) patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins) whose disease has progressed on or after platinum-based
Drugs
A 7/16-7/26/2024 FDA inspection at the Indoco Remedies drug manufacturing facility in Goa, India, found repeat significant violations of current good manufacturing practice (CGMP) regulations. A just-released 12/16/2024 Warning Letter says the specific violations were: * failure to record and justify any deviations from
Drugs
FDA has published a draft guidance, Obesity and Overweight: Developing Drugs and Biological Products for Weight Reduction, with recommendations to industry about developing drugs and biologics regulated by CDER intended for reduction and long-term maintenance of body weight in patients with obesity and those with a body mass index
Biologics
FDA has approved a guidance, Communications from Firms to Healthcare Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products: Questions and Answers, to reassure firms that if they provide communications that are consistent with the guidance’s recommendations, the agency will not consider the dissemination of such
Drugs
Johnson & Johnson is announcing positive topline results from the Phase 3 MARIPOSA study showing that its Rybrevant (amivantamab-vmjw) plus Lazcluze (lazertinib) had a statistically significant and clinically meaningful improvement in overall survival versus standard-of-care osimertinib (AstraZeneca’s Taggrisso) as a first-line therapy for patients with
Drugs
Two eukaryotic initiation factor 2B (eIF2B) activator drugs from Denali Therapeutics and Calico Life Sciences each failed to meet primary and secondary endpoints in an innovative platform trial studying their safety and effectiveness in treating amyotrophic lateral sclerosis (ALS; also known as Lou Gerig’s Disease), according to company statements.
Biologics
FDA has published a draft guidance, Study of Sex Differences in the Clinical Evaluation of Medical Products, with recommendations for increasing the number of females in clinical trials and non-interventional studies to help ensure the generalizability of results. The document says it also has recommendations for analyzing and interpreting
Drugs
FDA has approved Ionis’ Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA). An agency statement says FDA worked closely with Ionis during development to help design and implement the analysis upon which the approval was based. Spinraza’s efficacy was evaluated in
Drugs
FDA has posted a draft guidance entitled “Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway.” The document addresses the agency’s recent requirement that sponsors of an accelerated-approval drug/biologic must have their confirmatory trial underway at the time of approval. The requirement was mandated
Drugs
FDA has issued a draft guidance entitled “Considerations for Including Tissue Biopsies in Clinical Trials” that provides considerations for tissue biopsies in adults and children as part of clinical trials that evaluate investigational medical products. “Tissue biopsies in clinical trials are often needed to determine eligibility or to understand the
Drugs
FDA has issued a draft guidance entitled “Considerations for the Use of Artificial Intelligence (AI) to Support Regulatory Decision-Making for Drug and Biological Products.” An agency release says FDA has substantial experience in reviewing regulatory submissions with AI components, and they continue to exponentially increase. “AI can be used
Drugs
Neumora Therapeutics says its Phase 3 KOASTAL-1 Study of navacaprant for treating major depressive disorder (MDD) has “disappointing” results. The study did not show a statistically significant improvement regarding the primary endpoint of change from baseline in the Montgomery-Åsberg Depression Rating Scale total score at Week 6 or
Drugs
CDER approved 50 novel products in 2024, which was five (9%) less than the 55 products approved in 2023 and well above the 10-year rolling average of almost 46 approvals. Oncology approvals remained the top therapeutic category last year, with 15 (30%) novel approvals, according to the journal Nature.