achondroplasia
Nov 21, 2025
Achondroplasia Treatment: Emerging Therapies, Pathway Strategies, and Market Outlook
Achondroplasia, the most common form of dwarfism, is drawing increasing interest from pharmaceutical companies despite its rarity. Biomarin's VOXZOGO (vosoritide; a C-type natriuretic peptide; once-daily subcutaneous injection) is the only FDA-approved therapy for achondroplasia treatment. When it comes to BioMarin...
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Jun 03, 2025
Beam’s BEAM-302 Earns FDA Orphan Drug Tag for AATD; Incannex’s IHL-42X Moves to Phase III After FDA Protocol OK; Kura and Kyowa Kirin’s Ziftomenib Gets FDA Priority Review in NPM1-Mutant AML; GSK’s Linerixibat NDA Accepted by FDA for PBC-Linked Pruritus; Ascendis’ TransCon CNP Granted FDA Priority Review for Achondroplasia
Beam Therapeutics Secures FDA Orphan Drug Designation for BEAM-302 in AATD Beam Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to its investigational therapy, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a liver-targeting lipid nanoparticle (LNP)...
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Jan 22, 2024
Achondroplasia Treatment Space: Hunt for Potential Curative Therapies
Achondroplasia is a rare genetic disorder affecting bone growth, causing significant short stature, known as dwarfism. This condition is attributed to a fibroblast growth factor receptor 3 (FGFR3) gene mutation. The mutation results in an overactive FGFR3 gene, leading to a slowdown in bone formation within the car...
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Oct 24, 2023
FDA Approves PENBRAYA for Most Common Serogroups Causing Meningococcal Disease; BIMZELX Approved Moderate to Severe Plaque Psoriasis; FDA Approves BioMarin’s VOXZOGO; FDA Fast Track Designation to ANPD001 for Parkinson’s Disease; UCB Announces FDA Approval of ZILBRYSQ; EMA Granted Orphan Drug Designation to Lisata’s LSTA1
UCB announces FDA approval of ZILBRYSQ for the Treatment of Adults with Generalized Myasthenia Gravis On the 17th of October 2023, UCB (Euronext Brussels: UCB) made an announcement regarding the approval of ZILBRYSQ® (zilucoplan) by the US FDA for the management of generalized myasthenia gravis (gMG) in adult pa...
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