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FDA Breaks Precedent with Literature-Based Approval for Leucovorin in Cerebral Folate Deficiency

Drug ApprovalRare Disease
  • The FDA is initiating approval of leucovorin calcium tablets for cerebral folate deficiency based on systematic literature analysis rather than traditional clinical trials, marking a departure from the agency's standard evidence requirements.
  • The neurological condition affects folate transfer into the brain and causes developmental delays with autistic features, seizures, and movement coordination problems in patients.
  • President Trump referenced leucovorin as "a potential drug for some autism symptoms" during a recent media briefing, highlighting the connection between cerebral folate deficiency and autism spectrum features.
  • This approval represents a significant shift in FDA policy, as the agency has historically maintained that observational studies generate hypotheses but are insufficient for drug approvals.

Amneal Pharmaceuticals Receives FDA Approval for Generic Bimatoprost Eye Drops to Treat Glaucoma

Drug Approval
  • Amneal Pharmaceuticals received FDA approval for its generic version of bimatoprost ophthalmic solution 0.01%, a prostaglandin analog used to reduce elevated intraocular pressure in glaucoma patients.
  • The generic equivalent of LUMIGAN is available in three sizes (2.5 mL, 5 mL, and 7.5 mL) and addresses the growing need for affordable glaucoma treatments as prevalence rises among aging populations.
  • According to IQVIA data, the brand-name bimatoprost ophthalmic solution generated approximately $685 million in U.S. annual sales for the 12 months ended July 2025.
  • The approval represents a key growth driver for Amneal's Affordable Medicines segment, which focuses on complex generic products including injectables and biosimilars.

Federal Circuit Rules "Clinically Proven Effective" Claims Cannot Rescue Anticipated Drug Methods from Prior Art

Drug Approval
  • The Federal Circuit held that Bayer's patent claims for rivaroxaban and aspirin combination therapy were unpatentable despite including "clinically proven effective" language, ruling this limitation was functionally unrelated to the actual treatment method.
  • The court applied the functional relationship test from King Pharmaceuticals, determining that clinical validation does not transform an otherwise anticipated method when specific dosages are already disclosed in prior art.
  • This decision addresses concerns about patent "clawback" strategies where companies attempt to extend protection over known methods by adding post-hoc validation requirements that don't change how the treatment actually works.

FDA Issues Complete Response Letter for Scholar Rock's Apitegromab Due to Manufacturing Issues

Drug Approval
  • The FDA issued a Complete Response Letter declining approval of Scholar Rock's apitegromab for spinal muscular atrophy treatment due to manufacturing facility inspection findings.
  • The rejection was based on routine inspection issues at third-party manufacturer Catalent Indiana LLC, not related to the drug's safety or efficacy profile.
  • Scholar Rock plans to resubmit the Biologics License Application once Catalent Indiana addresses the FDA's manufacturing observations.
  • Company shares dropped approximately 15% in premarket trading following the regulatory setback announcement.

Shilpa Medicare Receives EMA Approval for Generic Rivaroxaban Orodispersible Films

Drug Approval
  • Shilpa Medicare Limited has secured European Medicine Agency approval for its generic Rivaroxaban Orodispersible Films, a bioequivalent alternative to Bayer's Xarelto tablets.
  • The oral dissolving film formulation offers enhanced patient comfort, particularly benefiting geriatric patients who may have difficulty swallowing traditional tablets.
  • This approval positions Shilpa Medicare to compete in Europe's $2.5 billion oral Rivaroxaban market with the only orodispersible film version of the anticoagulant.
  • The product represents the third prescription oral dissolving film approval from Shilpa's Bengaluru facility, which holds regulatory approvals from USFDA, EMA, and MHRA UK.

FDA Authorizes CytoCell KMT2A Companion Diagnostic for Revumenib in Acute Leukemia

Drug Approval
  • The FDA has granted De Novo Classification Request for OGT's CytoCell KMT2A Breakapart FISH Probe Kit PDx as a companion diagnostic for revumenib (Revuforj) in KMT2A-rearranged acute leukemia.
  • The diagnostic test rapidly identifies patients eligible for treatment with the first-in-class menin inhibitor revumenib, which was FDA-approved in November 2024 for relapsed/refractory acute leukemia.
  • More than 95% of patients with KMT2A-rearranged acute leukemia have a KMT2A translocation, making this diagnostic crucial for precision oncology in this high-risk patient population.
  • The companion diagnostic received Class II device classification rather than the more common Class III, reflecting OGT's extensive experience in hematology diagnostics and regulatory expertise.

A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

NCT04065399RecruitingPhase 1
Syndax Pharmaceuticals
Posted 11/5/2019

FDA Approves First Subcutaneous PD-1 Inhibitor as MSD Launches Keytruda QLEX

Drug ApprovalOncology
  • MSD secured FDA approval for Keytruda QLEX, the first subcutaneous PD-1 blocker, covering 38 solid tumor indications including NSCLC, triple-negative breast cancer, and melanoma.
  • The subcutaneous formulation offers faster administration in minutes versus hours for IV infusion, providing patients more treatment location choices and enhanced convenience.
  • Despite the approval, analysts remain skeptical about market impact due to combination therapy limitations and the looming 2028 patent cliff for Keytruda.
  • MSD projects 30-40% of patients will switch to the subcutaneous version within two years, with sales expected to exceed $1 billion by 2026 in NSCLC alone.

A Study to Evaluate the Safety and Efficacy of Pembrolizumab (MK-3475) Coformulated With Berahyaluronidase Alfa (MK-3475A) in Participants With Relapsed or Refractory Classical Hodgkin Lymphoma (rrcHL) or Relapsed or Refractory Primary Mediastinal Large B-cell Lymphoma (rrPMBCL)(MK-3475A-F65)

NCT06504394RecruitingPhase 2
Merck Sharp & Dohme LLC
Posted 10/14/2024

European Commission Publishes New Variations Guidelines to Streamline Drug Lifecycle Management

Drug Approval
  • The European Commission has published new Variations Guidelines, developed with EMA support, to streamline the lifecycle management of medicines and improve regulatory efficiency across the European Union.
  • The guidelines introduce a risk-based classification system for variations, categorizing changes as Type IA (minor changes with minimal impact), Type IB (minor changes requiring notification), or Type II (major changes like new indications).
  • The new framework will apply to variation applications submitted to EMA starting January 15, 2026, with updated procedural guidance to be published by December 2025.
  • These changes respond to increasing variation submissions driven by scientific and technological advances in medicine development, aiming to facilitate faster delivery of medicines to EU patients.

CROSSJECT Receives Additional $11.3M BARDA Funding for ZEPIZURE Needle-Free Epilepsy Treatment

Drug Approval
  • CROSSJECT secured an additional $11.3 million from BARDA, bringing total funding for ZEPIZURE development to $43.3 million.
  • The needle-free ZENEO Midazolam auto-injector is advancing toward FDA Emergency Use Authorization and New Drug Application submissions.
  • Upon FDA approval, BARDA will purchase 360,000 adult and pediatric autoinjectors for $60.8 million for the U.S. Strategic National Stockpile.
  • The ZENEO platform enables untrained caregivers to deliver emergency epilepsy treatment through clothing without needles.

Tumor-Infiltrating Lymphocytes and Macrophages Drive Cancer Immunoediting Through Three Critical Phases

Drug Approval
  • Cancer immunoediting progresses through three distinct phases—elimination, equilibrium, and escape—where tumor-infiltrating lymphocytes (TILs) and tumor-associated macrophages (TAMs) play opposing roles in tumor progression.
  • During the elimination phase, anti-tumor immune cells including CD8+ T cells, NK cells, and M1 macrophages actively destroy newly transformed cancer cells through cytotoxic mechanisms and pro-inflammatory signaling.
  • In the escape phase, immunosuppressive cells like regulatory T cells, M2 macrophages, and B-regulatory cells create a tumor-promoting microenvironment that facilitates immune evasion and metastasis.
  • Novel immunotherapeutic approaches targeting these immune cell populations, including TIL therapy, CAR-T cells, and macrophage repolarization strategies, show promise for overcoming tumor immune escape mechanisms.

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