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Budget Impact Analysis Shows Revumenib Cost-Neutral for Health Plans Despite High Specialty Drug Costs

Targeted TherapyRare Disease
  • A recent budget impact analysis demonstrates that revumenib (Revuforj) for relapsed/refractory acute leukemias with KMT2A translocation would be cost-neutral for health plans over three years.
  • The cost neutrality is primarily driven by revumenib's oral administration, which eliminates expensive infusion-related costs including chair time and administration fees.
  • The analysis highlights challenges in evaluating rare disease treatments affecting only 1.7 individuals per million patients, where traditional cost-effectiveness methods may not adequately capture therapeutic value.
  • Patient adherence to the oral therapy remains a critical variable that could influence long-term cost-effectiveness and clinical outcomes.

Longeveron Appoints Than Powell as Chief Business Officer to Lead Alzheimer's and HLHS Program Strategy

Rare Disease
  • Longeveron Inc. has appointed Than Powell as Chief Business Officer effective July 7, 2025, to oversee partnering efforts for Alzheimer's disease and international strategy for hypoplastic left heart syndrome programs.
  • The appointment comes as the company recently completed enrollment in its pivotal Phase 2 trial for laromestorcel in HLHS, with top-line data expected in approximately 13 months.
  • Powell brings over 25 years of pharmaceutical and biotech leadership experience from organizations including GSK and Eli Lilly, positioning the company for potential BLA submission.
  • Laromestorcel has received five FDA designations across its programs, including Orphan Drug, Fast Track, and RMAT designations for various indications.

Cellarity Initiates Phase 1 Trial of CLY-124, First-in-Class Oral Therapy for Sickle Cell Disease

AIRare Disease
  • Cellarity has dosed the first patient in a Phase 1 clinical trial of CLY-124, a novel oral medicine that increases fetal hemoglobin through a globin-switching mechanism to treat sickle cell disease.
  • The drug was discovered using AI-powered transcriptomic analysis and demonstrated the ability to increase fetal hemoglobin above 20% in preclinical studies without cytotoxicity.
  • CLY-124 represents a potential breakthrough for sickle cell disease treatment, offering a once-daily oral alternative to current therapies that carry dose-limiting toxicity.
  • The Phase 1 global trial will evaluate safety, tolerability, and pharmacokinetics in healthy volunteers before advancing to sickle cell disease patients.

GenSight Biologics Successfully Transfers LUMEVOQ Manufacturing to Catalent for LHON Gene Therapy

Rare Disease
  • GenSight Biologics has successfully transferred the upstream manufacturing process for LUMEVOQ, its gene therapy for Leber Hereditary Optic Neuropathy (LHON), to manufacturing partner Catalent Inc.
  • The partnership is expected to improve yield and upgrade analytical methods ahead of clinical use and regulatory submissions for the rare mitochondrial disease treatment.
  • Catalent manufactured a drug product batch released as safe for human use in November 2024, which will supply France's named patient early access program and dose-ranging study.
  • The collaboration positions GenSight for its planned global Phase III RECOVER trial beginning in 2026 and upcoming regulatory submissions to FDA and EMA.

XEOMIN Receives First-Ever Approval in Japan for Chronic Sialorrhea Treatment

Drug ApprovalRare Disease
  • Teijin Pharma has received approval from Japan's Ministry of Health, Labor and Welfare for XEOMIN (incobotulinumtoxinA) to treat chronic sialorrhea, marking the first drug approved for this indication in Japan.
  • The approval was based on Phase III clinical trials conducted by Merz Therapeutics in Germany and Poland, as well as trials conducted by Teijin in Japan.
  • XEOMIN works by inhibiting acetylcholine release from cholinergic nerve endings, effectively suppressing saliva secretion and reducing water and electrolyte secretion from salivary glands.
  • Chronic sialorrhea significantly impacts patients' quality of life, causing social isolation and difficulties with speaking and eating, while placing heavy burdens on families and caregivers.

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Episodic Migraine

NCT07018700RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Chronic Migraine

NCT07018713RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

FDA Approves Enhanced Labeling for Endari Based on Post-Marketing Pharmacokinetic Data

Drug ApprovalRare Disease
  • Emmaus Life Sciences received FDA approval for enhanced labeling of Endari (L-glutamine oral powder) based on additional post-marketing pharmacokinetic study data.
  • The updated label provides more comprehensive prescribing information, including confirmation of body weight-based dosing, no unwanted accumulation with twice-daily dosing, and flexible administration with or without food.
  • Endari remains approved to reduce acute complications of sickle cell disease in adult and pediatric patients five years and older, addressing a condition affecting approximately 100,000 people in the United States.
  • The label enhancement represents a significant improvement in clinical guidance for healthcare providers treating sickle cell disease patients with this prescription-grade L-glutamine therapy.

Phase 3 Trial Demonstrates Selumetinib Efficacy in Adults with Neurofibromatosis Type 1

Rare Disease
  • The KOMET phase 3 trial published in The Lancet shows selumetinib significantly reduces tumor size in one in five adults with neurofibromatosis type 1 plexiform neurofibromas.
  • This represents the first effective pharmacological treatment for adult NF1 patients, who previously relied solely on complex surgical interventions with limited success.
  • The randomized, double-blind study included 145 adult participants worldwide and demonstrated rapid pain reduction alongside decreased need for analgesics.
  • The innovative trial design allowed placebo participants to access selumetinib under specific conditions, enabling comprehensive efficacy assessment across different treatment timelines.

Ovid Therapeutics Sells Ganaxolone Royalty Rights to Immedica Pharma for $7 Million

Rare Disease
  • Ovid Therapeutics has entered into a definitive agreement with Immedica Pharma AB to sell its future royalty rights for ganaxolone sales outside China for $7 million in cash.
  • The transaction provides non-dilutive funding to support Ovid's ongoing operations, while Immedica strengthens its focus on ganaxolone by acquiring additional intellectual property rights.
  • Ganaxolone is an approved medicine in the EU, Great Britain, US, and China for treating epileptic seizures associated with CDKL5 deficiency disorder in patients aged 2-17 years.
  • Immedica will also assume financial responsibility for all costs related to the licensed intellectual property portfolio and plans to expand the license to include additional indications.

Former Pfizer CSO Mikael Dolsten Joins Two Biotech Boards, Advancing Gene Editing and Mast Cell Therapeutics

Gene EditingRare Disease
  • Dr. Mikael Dolsten, former Pfizer Chief Scientific Officer, has joined the boards of Arbor Biotechnologies and MC Sciences as both companies advance novel therapeutic platforms.
  • Dolsten's 16-year tenure at Pfizer included advancing over 150 drug candidates into clinical studies and leading regulatory approval of 36 medicines and vaccines.
  • Arbor Biotechnologies is progressing its lead gene editing program ABO-101 for primary hyperoxaluria type 1 into clinical trials.
  • MC Sciences is developing first-in-class mast cell-targeting therapeutics for diseases including chronic urticaria, systemic mastocytosis, and asthma.

Neuren Pharmaceuticals Secures US Patent for NNZ-2591 in Pitt Hopkins Syndrome Treatment

Orphan DrugRare Disease
  • Neuren Pharmaceuticals received US patent approval for NNZ-2591 to treat Pitt Hopkins syndrome, with patent protection extending to April 2040.
  • Phase 2 clinical trial results showed 9 out of 11 children demonstrated clinician-assessed improvements in communication, social interaction, cognition and motor abilities.
  • The FDA has granted both Orphan Drug and Fast Track designations for NNZ-2591 in PTHS, while no approved treatments currently exist for this debilitating condition.
  • Neuren also secured Rare Pediatric Disease Designation for NNZ-2591 in Phelan-McDermid syndrome, potentially qualifying for a priority review voucher.

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