Experimental Gene Therapy for Fanconi Anemia Earns Fast Track and Regenerative Medicine Advanced Therapy Designation

According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company’s investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designation as a treatment for Fanconi anemia from the US Food and Drug Administration (FDA). These designations were given based on encouraging data from ongoing clinical trials of the drug.

About Fanconi Anemia

Fanconi anemia is a rare genetic disease which is most characterized by a deficient response to DNA damage. Research surrounding this rare disorder has helped further the understanding of cancer development and the function of bone marrow. The disease is caused by genetic mutations affecting the protein normally responsible for DNA repair. Symptoms of Fanconi anemia include developmental abnormalities and deficiencies, short stature, fatigue, pale skin, easy bruising, infections, and acute myeloid leukemia. Bone marrow failure means that the body cannot produce blood cells. Treatment may include androgens and hematopoietic growth factors. A stem cell transplant can permanently cure the condition. The risk of serious complications and cancer means that many patients do not survive into their adult years. Fanconi anemia commonly appears alongside other genetic conditions and is more prevalent in the Ashkenazi Jewish population. To learn more about Fanconi anemia, click here.

About RMAT Designation

RMAT designation is intended specifically for regenerative gene and cell therapies that are designed to cure, reverse, or otherwise treat a serious, life threatening disease. To qualify, a drug must display potential in the clinical setting to address a currently unmet medical need. Drugs that earn RMAT designation will be subject to more close collaboration between the FDA and the developer which will increase the likelihood of successful approval.

About Fast Track Designation

RMAT designation also carries all of the benefits of Fast Track and Breakthrough Therapy designations. Fast Track designation is intended for experimental therapies that address a life threatening disease and fulfill a medical need that is not being addressed by currently available therapies. The recipient company will be able to work more closely with the FDA during the drug’s development.

Rocket intends to have clinical trials of RP-L102 begin early in 2019. This trial aims to involve 12 patients with Fanconi anemia.


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