Healthcare Industry News: Crigler-Najjar syndrome
News Release - June 5, 2018
Audentes Announces Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to AT132 for the treatment of X-Linked Myotubular MyopathySAN FRANCISCO, June 5, 2018 -- (Healthcare Sales & Marketing Network) -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM). The PRIME designation is intended to enhance interaction and early dialogue with developers of promising therapies to optimize development plans and speed evaluation so that these medicines may reach patients as early as possible.
"We are pleased that AT132 has been accepted into the PRIME program, which is analogous to the Breakthrough Therapy and Regenerative Medicine and Advanced Therapy designations from the U.S. Food and Drug Administration," stated Mary S. Newman, Senior Vice President, Regulatory Affairs. "This PRIME designation has been enabled by the positive interim data reported from the first dose cohort of ASPIRO, the Phase 1/2 clinical trial of AT132, and we look forward to collaborating with the EMA to accelerate the clinical development of this potentially transformative therapy for XLMTM patients."
About PRIME Designation
The PRIME program was launched by the EMA in March 2016, and the designation is designed to aid and expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. The PRIME designation provides appointment of a rapporteur, early dialogue and scientific advice at key development milestones, and the potential to qualify products for accelerated review earlier in the application process.
About AT132 for X-Linked Myotubular Myopathy
AT132 is the Audentes product candidate being developed to treat XLMTM, a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age. XLMTM is caused by mutations in the MTM1 gene, which encodes the protein myotubularin. Myotubularin plays an important role in the development, maintenance and function of skeletal muscle cells. AT132 is comprised of an AAV8 vector containing a functional copy of the MTM1 gene. In May 2018, Audentes reported positive interim data from the first dose cohort of ASPIRO, a multicenter, ascending dose Phase 1/2 clinical study to evaluate the safety and preliminary efficacy of AT132 in approximately 12 XLMTM patients less than five years of age. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).
AT132 has been granted PRIME and Orphan Drug designations by the EMA, and Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA.
About Audentes Therapeutics, Inc.
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases. We are currently conducting Phase 1/2 clinical studies of our lead product candidates AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM) and AT342 for the treatment of Crigler-Najjar syndrome. We have two additional product candidates in development, including AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT). We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.
For more information regarding Audentes, please visit www.audentestx.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of the company to work with the EMA to accelerate the clinical development and potential approval of AT132. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercial its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.
Source: Audentes Therapeutics
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