October 2, 2019

NS Pharma Completes Rolling NDA Submission to FDA for NS-065/NCNP-01 (viltolarsen)

We are pleased to learn that NS Pharma has completed the submission of its rolling New Drug Application (NDA) to the FDA for NS-065/NCNP-01 (viltolarsen), which is under development for the treatment of Duchenne amenable…

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September 16, 2019

Wave Life Sciences Announces Fast Track Designation from U.S. FDA for Suvodirsen

Wave Life Sciences shared today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to suvodirsen for the treatment of Duchenne in patients amenable to exon 51 skipping. Fast Track designation…

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September 5, 2019

Sarepta Therapeutics Shares Summer 2019 Newsletter

Sarepta has shared the Summer 2019 issue of their newsletter, including clinical updates. Click here to download.

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August 22, 2019

WATCH: Community Discussion with Sarepta Therapeutics – Webinar Recording

Yesterday PPMD invited Sarepta leaders to join a community discussion regarding the New Drug Application for golodirsen. We apologize if you were unable to access the webinar audio due to the high number of attendees….

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August 19, 2019

Sarepta Therapeutics Receives Complete Response Letter from the FDA for Golodirsen New Drug Application

PPMD is very disappointed to learn that Sarepta Therapeutics received a Complete Response Letter from the FDA regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne in…

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August 16, 2019

ICER Releases Report; PPMD Issues Open Letter to Payers

Last night, the Institute for Clinical and Economic Review (ICER) released their final report assessing three Duchenne products: Emflaza, EXONDYS 51, and golodirsen. This report integrates all public comments, stakeholder engagement, and the Roundtable proceedings…

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August 7, 2019

Audentes Therapeutics Announces Plans to Commence Patient Dosing with AT702 in Q4 2019

Audentes Therapeutics has announced plans to commence patient dosing in the fourth quarter of 2019 with AT702 produced by Nationwide Children’s Hospital. AT702 is designed to induce exon 2 skipping to treat Duchenne caused by…

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April 16, 2019

Wave Life Sciences Announces Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial Design

Wave Life Sciences shared safety and tolerability data from their Phase 1 clinical trial evaluating investigational suvodirsen (WVE-210201) in patients with Duchenne. The results from this trial support progressing to a Phase 2/3 clinical trial,…

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April 8, 2019

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

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March 28, 2019

Sarepta Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of ESSENCE Study

Sarepta Therapeutics announced positive results from its interim analysis of muscle biopsies at week 48, comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, randomized double-blind,…

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