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Solid Biosciences Receives FDA and Health Canada Approval for First-in-Class Gene Therapy Targeting Fatal Heart Rhythm Disorder

Orphan DrugRare Disease
  • Solid Biosciences has received FDA IND and Health Canada CTA approval for SGT-501, a novel gene therapy designed to treat catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare genetic heart condition that can cause sudden death.
  • SGT-501 represents the first potential therapy to address the underlying mechanisms of CPVT by delivering a functional copy of the CASQ2 gene to stabilize calcium regulation in heart muscle cells.
  • The company plans to initiate a Phase 1b clinical trial in the fourth quarter of 2025, marking a significant milestone for patients with this life-threatening condition that currently has no approved treatments.
  • CPVT affects approximately 1 in 10,000 individuals globally and is often triggered by physical activity or emotional stress, leading to dangerous arrhythmias in otherwise healthy hearts.

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

NCT06138639RecruitingPhase 1
Solid Biosciences Inc.
Posted 5/6/2024

Recursion Pharmaceuticals Acquires Full Rights to REV102, Potential First-in-Class Oral ENPP1 Inhibitor for Hypophosphatasia

Rare Disease
  • Recursion Pharmaceuticals acquired full rights to REV102, a potential first-in-class oral ENPP1 inhibitor for hypophosphatasia (HPP), from joint venture partner Rallybio for up to $25 million.
  • REV102 targets ENPP1 enzyme to restore inorganic pyrophosphate balance for proper bone mineralization, potentially offering the first oral disease-modifying therapy for HPP patients.
  • The drug candidate is currently in IND-enabling studies with Phase 1 clinical trials expected to begin in the second half of 2026.
  • HPP affects over 7,800 diagnosed patients across the US and EU5, with many having limited access to current enzyme replacement therapies.

Hansa Biopharma Appoints Dr. Richard Philipson as Chief Medical Officer to Lead Next Development Phase

Rare Disease
  • Hansa Biopharma has appointed Dr. Richard Philipson as Chief Medical Officer effective July 14, bringing over 25 years of industry experience to the commercial-stage biopharmaceutical company.
  • Dr. Philipson's track record includes clinical leadership resulting in four product approvals, including rare disease and gene therapy treatments, with expertise spanning first-in-human to Phase 3 studies.
  • The appointment positions Hansa to advance its proprietary IgG-cleaving enzyme technology platform, including imlifidase for kidney transplantation and HNSA-5487 for autoimmune diseases.
  • Dr. Philipson will support upcoming regulatory submissions and help build strategic development plans for future indications across rare diseases, inflammation, and oncology.

Taiho's Pizuglanstat Fails to Meet Primary Endpoint in Phase III Duchenne Muscular Dystrophy Trial

Rare Disease
  • Taiho Pharmaceutical's pizuglanstat (TAS-205) failed to demonstrate significant improvement in time to rise from floor compared to placebo in the Phase III REACH-DMD study.
  • The randomized, double-blind trial enrolled 82 male DMD patients aged 5 years and older across 26 Japanese sites over three years.
  • This setback adds to recent DMD therapeutic failures, including Sarepta/Roche's Elevidys and Pfizer's withdrawn gene therapy program.
  • Pizuglanstat works by inhibiting hematopoietic prostaglandin D synthase to reduce muscle necrosis and inflammatory response in DMD patients.

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）

NCT04587908Active, Not RecruitingPhase 3
Taiho Pharmaceutical Co., Ltd.
Posted 11/1/2020

FDA Authorizes Emergency Compassionate Use of VAR 200 for Ultra-Rare ApoCII Amyloidosis

Rare DiseasePrecision MedicineOrphan Drug
  • The FDA has authorized emergency compassionate use of ZyVersa Therapeutics' VAR 200 for a patient with ApoCII amyloidosis, an ultra-rare kidney disease with no effective treatment options.
  • VAR 200, currently in Phase 2a trials for diabetic kidney disease, targets lipid accumulation in kidneys and was selected based on evidence of lipotoxic glomerular injury in the patient's kidney biopsy.
  • The treatment will be administered at the University of Miami under Dr. Alessia Fornoni's care, following the protocol of the ongoing diabetic kidney disease clinical trial.
  • This compassionate use case represents a precision medicine approach to match patients with specific molecular signatures to targeted therapies for rare diseases.

P2a Open Label Study to Evaluate 2-HPβCD in Subjects With Diabetic Kidney Disease

NCT06489340RecruitingPhase 2
ZyVersa Therapeutics, Inc.
Posted 6/11/2025

Lobe Sciences Secures European Patent Protection for DHA-Based Sickle Cell Disease Treatment

Rare DiseaseRare Disease
  • Lobe Sciences has obtained European Unitary Patent EP4117446 validation, providing intellectual property protection across 17 EU member states for its DHA and egg yolk-based composition targeting Sickle Cell Disease.
  • The patent covers the formulation underlying Altemia® Medical Food, a first-in-class emulsified oral treatment designed to restore red blood cell membrane integrity and address long-chain fatty acid deficiencies in SCD patients.
  • This validation strengthens Lobe's intellectual property portfolio and enables broader commercialization of Altemia® across key European markets as part of the company's global expansion strategy.

OMass Therapeutics to Present First Preclinical Data for MC2 Receptor Antagonist OMS1620 at ENDO 2025

Rare Disease
  • OMass Therapeutics will present the first public preclinical data for OMS1620, a potential best-in-class MC2 receptor antagonist targeting congenital adrenal hyperplasia (CAH).
  • OMS1620 is designed to maximize receptor residency time, making it highly resistant to competition from rising endogenous ACTH levels as glucocorticoid doses are reduced.
  • The development candidate is currently in IND enabling studies and aims to help CAH patients achieve androgen normalization while on physiological glucocorticoid replacement doses.
  • The poster presentation will take place at ENDO 2025 in San Francisco on July 13, marking a significant milestone for the company's lead program.

FUJIFILM's FF-10832 Receives FDA Orphan Drug Designation for Biliary Tract Cancer Treatment

OncologyRare Disease
  • The FDA has granted orphan drug designation to FF-10832, FUJIFILM's investigational liposomal formulation of gemcitabine, for treating biliary tract cancer.
  • Phase 1 study results presented at ASCO 2025 demonstrated that FF-10832 is well tolerated and shows anti-tumor activity in patients with advanced biliary tract cancer.
  • The novel liposomal formulation is designed to enhance anti-tumor activity by prolonging plasma half-life and improving targeted delivery to tumors.
  • Biliary tract cancer affects approximately 16,000 new patients annually in the U.S., with most presenting with unresectable or metastatic disease and poor survival rates.

A Phase 1 Dose-escalation Study of FF-10832 for Treatment of Solid Tumors Including Biliary Tract Cancer

NCT03440450Active, Not RecruitingPhase 1
Fujifilm Pharmaceuticals U.S.A., Inc.
Posted 3/22/2018

A Study to Evaluate Safety, Efficacy of FF-10832 in Combo With Pembrolizumab in Urothelial & Non-small Cell Lung Cancer

NCT05318573RecruitingPhase 2
Fujifilm Pharmaceuticals U.S.A., Inc.
Posted 6/1/2022

Bezuclastinib Achieves 87% Response Rate in Pivotal Trial for Non-Advanced Systemic Mastocytosis

Rare DiseasePrecision MedicineOncology
  • Cogent Biosciences' bezuclastinib demonstrated superior symptom improvement with a placebo-adjusted difference of 8.91 points in total symptom score at 24 weeks (p=0.0002) in the SUMMIT trial.
  • The drug showed remarkable efficacy on mast cell burden, with 87.4% of patients achieving at least 50% reduction in serum tryptase compared to 0% in the placebo group.
  • Bezuclastinib exhibited a favorable safety profile with most adverse events being low-grade, supporting its potential for chronic use in systemic mastocytosis patients.
  • The company plans to submit a New Drug Application to the FDA by the end of 2025 based on these positive results from the registration-directed trial.

(Summit) a Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients with Indolent or Smoldering Systemic Mastocytosis

NCT05186753Active, Not RecruitingPhase 2
Cogent Biosciences, Inc.
Posted 6/27/2022

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