🧬 This Week in Rare Diseases: FDA Approvals, Global Partnerships, Breakthrough Therapies, and More
This week’s Rare Diseases roundup brings you pivotal updates on new FDA approvals, groundbreaking partnerships, and critical advancements in treatments for rare diseases. From promising orphan drug designations to urgent access to life-saving therapies, these insights will keep you at the forefront of the rare disease landscape.
🔬 Key highlights:
🌍 Sanofi’s Rilzabrutinib Earns Orphan Designation for IgG4-RD in the EU
A significant step forward in the treatment of IgG4-related disease, Sanofi’s rilzabrutinib has received orphan drug designation from the EMA, following positive Phase 2 results.
🎥 FDA Grants Orphan Drug Status to Dusquetide for Behçet’s Disease
Soligenix’s Dusquetide gains FDA orphan drug status, backed by encouraging Phase 2a data showing improvement in oral ulcers, a key symptom of Behçet’s disease.
🇮🇳 India’s Goa Launches Pricing Policy for Cancer & Rare Disease Treatments
Goa introduces a new pricing policy to improve affordability and access to critical cancer and rare disease therapies in the region.
🚨 NORD Announces $245K Seed Grants to Accelerate Rare Disease Research
NORD pledges $245,000 in grants to fund early-stage research, supporting innovative studies on rare conditions like Epidermodysplasia Verruciformis and Pseudomyxoma Peritonei.
🧬 FDA Approves Brensocatib for Non-Cystic Fibrosis Bronchiectasis
Brensocatib becomes the first FDA-approved treatment for non-cystic fibrosis bronchiectasis, a breakthrough for chronic lung disease patients.
🧠 Biohaven Ends Development of Troriluzole in OCD After Phase 3 Failure
Biohaven halts the development of Troriluzole for OCD after Phase 3 trial failure, refocusing efforts on other promising pipeline programs.
👁️ Sarepta Shares Elevidys Safety Data Amid Petition for Label Update
Sarepta shares new safety data for Elevidys, its gene therapy for Duchenne muscular dystrophy, in response to an FDA petition seeking a label update.
💡 Atropos Health and Novartis Collaborate on AI to Speed Rare Disease Diagnosis
Atropos Health and Novartis partner to leverage AI for faster diagnosis of rare diseases, including paroxysmal nocturnal hemoglobinuria, improving patient outcomes.
🌟 National Psoriasis Foundation and Boehringer Ingelheim Launch GPP Awareness Day
The first-ever GPP Awareness Day raises awareness about generalized pustular psoriasis, a rare and life-threatening skin condition.
💰 Neurogene Reports Progress on NGN-401 for Rett Syndrome
Neurogene moves closer to a registrational phase for its gene therapy NGN-401, with updated clinical data expected later in 2025.
📢 Stay Ahead in Rare Disease Research!
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