DelveInsight’s, “Spinal Muscular Atrophy Pipeline Insight 2024” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Spinal muscular atrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Spinal Muscular Atrophy Pipeline Report
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Spinal Muscular Atrophy Emerging Drugs Profile
Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The US Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Currently, it is in phase III stage of development.
ACTX-401 is an AAV9-delivered gene replacement therapy for the treatment of a rare form of Spinal Muscular Atrophy (SMA) called SMA with Respiratory Distress type 1 (SMARD1), and for Charcot-Marie-Tooth type 2S (CMT2S). SMARD1 and CMT2S are autosomal recessive genetic disorders caused by mutations in the gene that produces immunoglobulin mu-binding protein 2 (IGHMBP2) and are part of a larger family of disorders called IGHMBP2-related disorders (IRDs). ACTX-401 delivers a functional copy of the IGHMBP2 gene to patients, restoring expression of functional IGHMBP2. ACTX-401 clinical success in SMARD1 and CMT2S could lead to applicability to other IRDs in the future. Currently, it is being investigated in Phase I/II stage of development for the treatment of spinal muscular atrophy.
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Spinal muscular atrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration
Spinal Muscular Atrophy Products have been categorized under various Molecule types such as
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Scope of the Spinal Muscular Atrophy Pipeline Report
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