• Regulatory NewsRegulatory News

    FDA to form task force, public-private partnership on rare neurodegenerative diseases

    The US Food and Drug Administration (FDA) released a five-year action plan for accelerating drug development for rare neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), as well as improving access to novel therapies.   “The effects of rare neurodegenerative diseases are devasting, with very few effective therapeutic options available to patients,” Robert M. Califf, MD, FDA Commissioner, said in a statement. “This action plan, especially including ...
  • This Week at FDAThis Week at FDA

    This Week at FDA: MDUFA’s turn in Congress, another COVID vaccine adcomm, and more

    Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation and what we’re reading from around the web. This week, we learn that the House Energy and Commerce committee will finally take up legislation to reauthorize the Medical Device User Fee Amendments (MDUFA) next Wednesday. Plus, we look at upcoming developments on COVID-19 vaccines and a much-anticipated advisory committee meeting o...
  • Regulatory NewsRegulatory News

    FDA Finalizes ALS Drug Development Guidance

    The US Food and Drug Administration (FDA) on Monday finalized guidance to assist in the clinical development of treatments for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The 11-page guidance, which finalizes a draft version from February 2018, includes information on general development, safety and effectiveness considerations, as well as specific effectiveness trial considerations. The guidance incorporates parts of a guidance put toget...
  • Regulatory NewsRegulatory News

    Final ALS Drug Development Guidance Coming Soon

    Acting US Food and Drug Administration (FDA) Commissioner Ned Sharpless on Thursday announced that the agency would finalize guidance on developing treatments for amyotrophic lateral sclerosis (ALS) by the end of September. The 9-page draft guidance, first released in February 2018 , incorporated parts of a draft guidance put together by the ALS Association, known for its viral "ice bucket challenge," which FDA said provided the agency "with scientific advice and insig...
  • Regulatory NewsRegulatory News

    Draft and Final FDA Guidances Detail Development of New Drugs for Neurological Disorders

    The US Food and Drug Administration (FDA) on Thursday released seven draft, revised draft and final guidance documents on everything from how to develop new drugs to treat early Alzheimer’s, Duchenne Muscular Dystrophy and acute migraines to guidance on how to prepare a pre-request for designation for a combination product. FDA Commissioner Scott Gottlieb noted the five guidances issued to address neurological disorders "benefited from the streamlined approach" of a pi...
  • Meeting to Focus on Regulatory Paradigm of Developing, Evaluating ALS Therapies

    The US Food and Drug Administration (FDA) will soon hold a public meeting to obtain input on ways to enhance the development of new therapies to treat amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease. The condition affects patients by degenerating their nerve cells, causing a loss of muscle control over some of the body's key functions, including breathing and swallowing, ultimately causing death. At present, there is no known cure for the dise...
  • For Terminally Ill Patients, 'Life Spans Shorter Than FDA's Approval Process' Spurs DIY Medicine

    Some terminally ill patients are turning research and development into a DIY enterprise, frustrated at the pace of getting potentially life-saving treatments through the US Food and Drug Administration (FDA), reports The Wall Street Journal . "We simply don't have time to wait for the results of [clinical trials]," said Ben Harris, who suffers from Lou Gehrig's Disease (ALS), to the Journal . "Our life spans are much shorter than the [Food and Drug Administration] appr...