Regulatory environment for novel therapeutic development in mitochondrial diseases.

ABSTRACT: At present, there is just one approved therapy for patients with mitochondrial diseases in Europe, another in Japan, and none in the United States. These facts reveal an important and significant unmet need for approved therapies for these debilitating and often fatal disorders. To fill this need, it is critical for clinicians and drug developers to work closely with regulatory agencies. In the United States, mitochondrial disease patients and clinicians, the United Mitochondrial Disease Foundation, and pharmaceutical industry members have engaged with the Food and Drug Administration to educate each other about these complex and heterogeneous diseases and about regulatory requirements to obtain approvals for novel therapies. Clinical development of therapies for rare diseases has been fac

SUBMITTER: Hirano M 

PROVIDER: S-EPMC9326497 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature