PTC Therapeutics Announces FDA Grants Priority Review to Risdiplam for the Treatment of Spinal Muscular Atrophy
The NDA filing is based on 12-month data from the dose-finding portion of the pivotal FIREFISH and SUNFISH studies, and clinical and preclinical pharmacokinetic and pharmacodynamic data. FIREFISH is an open-label, two-part clinical trial of risdiplam in infants with SMA type 1. SUNFISH is a double-blind, two-part, placebo-controlled trial of risdiplam in patients with type 2 or 3 SMA aged 2-25 years. SUNFISH part 2 recently met its primary endpoint of change from baseline in the Motor Function Measure 32 scale. Results from the study will be presented at an upcoming medical congress. The SMA program is a collaboration between PTC, the
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that is the leading genetic cause of mortality in infants and toddlers caused by deletion or mutation in the survival of motor neuron 1 (SMN1) gene, which results in reduced levels of SMN protein. The related SMN2 pre-mRNA is alternatively spliced producing only small amounts of functional SMN protein. Insufficient levels of SMN protein result in the progressive loss of motor neurons leading to muscle atrophy and death in its most severe form. It is estimated that 1 in every 11,000 newborn children will develop SMA.
Risdiplam is an investigational medicine being studied in a broad range of patients with SMA from birth to 60 years of age. It is designed to provide sustained increase in SMN protein centrally and peripherally through daily dosing and is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body. Risdiplam is being studied in a clinical trial for patients with type 1 SMA, called FIREFISH, in pre-symptomatic babies, RAINBOWFISH, in patients who have been in previous clinical trials for SMA, JEWELFISH and in SUNFISH, a placebo-controlled study in people aged 2-25 years with Type 2 or 3 SMA.
About the SMA collaboration
The SMA program was initiated by
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
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This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; advancement of PTC's joint collaboration program in SMA, including any potential regulatory submissions, regulatory approvals or royalty or milestone payments; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
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As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including risdiplam.
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