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  • Regulatory NewsRegulatory News

    Sanders, Cummings Slam Marathon’s $89,000 Price Tag for Old Steroid

    Sen. Bernie Sanders (D-VT) and Rep. Elijah Cummings (D-MD) on Monday wrote to Illinois-based Marathon Pharmaceuticals CEO Jeffrey Aronin, calling the company’s plan to charge $89,000 annually for its recently approved treatment “outrageous.” Not only did Marathon not develop the steroid, known as Emflaza (Deflazacort) and approved by the US Food and Drug Administration last week to treat Duchenne Muscular Dystrophy, but the letter notes that the steroid is curren...
  • Regulatory NewsRegulatory News

    EMA Contests Two Judicial Decisions Over Clinical Trial Transparency Efforts

    The European Medicines Agency (EMA) on Thursday announced it has appealed two interim orders by the President of the General Court of the EU, which suspended the release of clinical study documents requested by third parties under a new transparency regulation. The first order blocked the release of a study report for PTC Therapeutics’ Translarna (ataluren), a Duchenne’s muscular dystrophy treatment, until a final ruling is provided. EMA says it was planning to provid...
  • Regulatory NewsRegulatory News

    Sarepta Wins Controversial FDA Approval for First DMD Drug

    The US Food and Drug Administration (FDA) on Monday approved Sarepta Therapeutics’ first drug to treat patients with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness in young children. The approval is highly controversial after a FDA advisory committee voted against approval in April as the outside experts said there was not substantial evidence that the drug is effective in providing clinical benefit...
  • Regulatory NewsRegulatory News

    Republican Senators Lobby FDA Ahead of Decision on Sarepta’s DMD Drug

    The contentious debate over whether the US Food and Drug Administration (FDA) will approve or deny Sarepta’s Duchenne Muscular Dystrophy (DMD) drug eteplirsen will come to an end either on or before Thursday of this week. But before that decision is made, two Republican senators, Ron Johnson (WI), Chairman of the Committee on Homeland Security and Governmental Affairs, and Dan Coats (IN), sent a letter to FDA Commissioner Robert Califf last Friday to “express disapp...
  • Regulatory NewsRegulatory News

    FDA Panel Votes Against Approval for Sarepta DMD Drug

    Following a contentious debate pitting young boys with the terminal illness Duchene Muscular Dystrophy (DMD) against the US Food and Drug Administration (FDA), an advisory panel of outside experts on Monday voted against the approval of Sarepta Therapeutics’ controversial investigational drug eteplirsen to treat DMD. The agency’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7-3 against approval, with three abstaining, and 7-6 against the accelerat...
  • Regulatory NewsRegulatory News

    Academic DMD Experts Criticize ‘Errors’ in FDA Ad Com Briefing Documents

    More than 35 medical professors and experts have criticized the US Food and Drug Administration’s (FDA) review of a Duchenne Muscular Dystrophy (DMD) drug, citing errors in the agency’s advisory committee meeting briefing documents. At issue is Sarepta’s drug eteplirsen, which the company is seeking approval as the world’s first treatment for DMD, a rare and fatal genetic disorder afflicting about 15,000 boys in the US. The drug was initially supposed to go before the Pe...
  • Regulatory NewsRegulatory News

    European Regulatory Roundup: Lilly May Move API Production Outside EU Due to REACH Regulations (17 March 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Eli Lilly Threatens to Move API Production out of the EU in Broadside Against REACH Eli Lilly has warned it may move production of certain active pharmaceutical ingredients (APIs) out of the European Union because of REACH regulations. The production of two of Lilly’s veterinary APIs is tied to the availability of cobalt salts, something it says is becoming a “significant pr...
  • Regulatory NewsRegulatory News

    FDA Faces Sensitive Challenge With Review of DMD Drug

    The blanketing of snow on the East Coast this past weekend prompted the postponement of a high-profile US Food and Drug Administration (FDA) advisory committee hearing for a Duchene Muscular Dystrophy (DMD) drug that is stirring up public controversy and will eventually lead to an up or down decision testing FDA’s resilience to public pressure. The advisory committee meeting, which the Jett Foundation, a DMD advocacy group, said Tuesday will likely be rescheduled withi...
  • Regulatory NewsRegulatory News

    FDA Releases Highly Anticipated Guidance on Duchenne Muscular Dystrophy

    A new draft guidance document released today by the US Food and Drug Administration (FDA) aims to clarify the development process for new drugs intended to treat Duchenne Muscular Dystrophy (DMD), a progressive, fatal disease for which there is currently no cure. Background DMD is a rare disease almost exclusively affecting young men. Due to a genetic defect, the body does not produce dystrophin, which ensures the proper function of muscle tissue. Over time, this resul...
  • Regulatory NewsRegulatory News

    FDA Wants Input on Patient-Developed DMD Guidance

    The US Food and Drug Administration (FDA) is calling for public comment on a new draft guidance document developed by patient advocates and intended to accelerate the development of therapies to treat Duchenne Muscular Dystrophy (DMD). Background In June 2014, Parent Project Muscular Dystrophy (PPMD) and other stakeholders submitted a draft guidance document to FDA which they said would "help accelerate development and review of potential therapies for DMD." The guid...
  • Feature ArticlesFeature Articles

    Filling a Regulatory Void: Patient Advocates Submit Guidance for Duchenne Muscular Dystrophy

    Muscular dystrophy (MD)––a group of diseases that cause progressive muscle weakness due to defects in muscle proteins ––has a range of complex features that make treatments elusive. MD exists in many forms (Duchenne MD is the most common); animal models do not accurately reflect human disease; and it is difficult to define and measure the progression of this slow disease. Because MD can be life-threatening, new disease-modifying (serving to slow or halt progression) an...