Photo from The Boston Globe 4/25/2016
Families with children with Duchenne muscular dystrophy (“DMD”) from all over the United States flew to Maryland to attend a Food and Drug Administration (“FDA”) advisory committee meeting on a mission to convince committee members to recommend accelerated approval of a new drug application for eteplirsen. Scientists argued that the clinical trials provided evidence that the drug slowed the progression of DMD. Parents received time-limited slots to discuss their own observations of the positive effects of the drug.
The accelerated approval provisions in Section 506(c) of FD&A Act, added by Section 112 of the Food and Drug Administration Modernization Act of 1997, allows for accelerated approval to “a product for a serious or life-threatening disease or condition . . . upon a determination that the product has an effect on a surrogate endpoint that is reasonable likely to predict clinical benefit, or on a clinic endpoint that can by measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments.”
The advisory committee was asked if the applicant provided substantial evidence from adequate and well-controlled studies that Eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit. The phrasing of the question is not applicable where the study participation is small due to the rarity of the disease, such as DMD. Surrogate endpoints and intermediate clinical endpoints should be considered in situations for clinical studies for drugs for rare diseases.
Unfortunately, after hours of testimony, the committee recommended against approval for Sarepta’s exon-skipping drug eteplirsen.
The FDA is allowed to expedite programs for serious conditions in Section 506(b) of the FD&C Act as added by Section 112 of the Food and Drug Administration Modernization Act of 1997 and amended by Section 901 of the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA). Receiving and acknowledging the testimony of patients is part of the drug review process under the Patient Focused Drug Developments initiative launched by FDASIA.
By utilizing the flexibility offered under FDASIA, the FDA can fast-track the approval process for Eteplirsen.
Every minute matters to families with boys who have the rare disease. The final FDA decision is with the director of the drug evaluation center, Janet Woodcock.
Debra Miler, co-founder and CEO of CureDuchenne, a nonprofit organization, offers her words of encouragement for approval at www.cureduchenne.org. The advancement of treatment for a child with DMD affects the quality of life.
Senator Rubio brought attention to this matter – click here for the video SenatorMarcoRubio.
