• Regulatory NewsRegulatory News

    GAO Raises Questions on FDA’s Orphan Drug Designation Process

    The Government Accountability Office (GAO) announced Friday that it has found that the US Food and Drug Administration (FDA) does not always ensure that all information is consistently recorded and evaluated when making orphan drug designation determinations. The missing of these critical steps by the agency in some cases means that FDA reviewers do not fully understand the context of a drug’s intended use for the rare disease, GAO said. “The Commissioner of FDA shou...
  • Regulatory NewsRegulatory News

    Protalex Wins $403K Grant from FDA Office of Orphan Products Development

    Clinical-stage biopharma Protalex, Inc. on Thursday said it has been awarded a $403,000 grant from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) to support future clinical development activity of PRTX-100 as a treatment for immune thrombocytopenia (ITP). The grant is part of a larger program that will award $23 million in research grants for 21 clinical trials over the next four years to boost the development of rare dise...
  • Regulatory NewsRegulatory News

    Bipartisan Group of Senators Introduces Bill to Speed Development of Rare Disease Drugs

    A new bipartisan bill aims to help accelerate the development of targeted drugs to treat rare diseases, including Duchenne muscular dystrophy, cystic fibrosis, some cancers and other genetic diseases. Although it's unclear how exactly the bill will aid in the development of targeted rare disease treatments, one of the cosponsors, Sen. Elizabeth Warren (D-MA), said the bill would clarify the US Food and Drug Administration's (FDA) current authority to consider resear...
  • FDA Guidance: How to Improve Your Meetings With the Office of Orphan Product Development

    A new draft guidance document published by the US Food and Drug Administration (FDA) aims to clarify the processes and procedures of meetings with its Office of Orphan Products Development (OOPD). Background OOPD's mission is to help facilitate the development of drugs, devices, biologics and other medical products to treat patients with rare diseases. Those diseases, as defined by the Orphan Drug Act of 1983, affect fewer than 200,000 patients in the US in any given ...