sickle cell disease Archives - BioPharma Global

Editas Medicine Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Beta Thalassemia

June 8, 2022

CAMBRIDGE, Mass., May 12, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of beta thalassemia. The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease.

“Beta thalassemia is a devastating disease that leads to severe anemia, organ failure, and premature death. Receiving Orphan Drug Designation for EDIT-301 for beta thalassemia highlights the urgent need for new treatment options for patients,” said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine. “Preparations to initiate the Phase 1/2 clinical trial of EDIT-301, a potentially transformative medicine for people living with beta thalassemia, are underway, and we look forward to dosing the first patient in the clinical trial this year.”

The FDA’s Orphan Drug Designation program provides orphan status to drugs or biologics intended for the prevention, diagnosis, or treatment of diseases that affect fewer than 200,000 people in the United States. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

Click here to read the full article at GlobeNewswire

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BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Graphite Bio Announces U.S. FDA Fast Track Designation Granted to GPH101 for the Treatment of Sickle Cell Disease

May 25, 2022

by administrator BPG Blog

“The FDA’s decision to grant Fast Track Designation to GPH101 for sickle cell disease signifies the need for novel medicines for this serious genetic disease and supports the ongoing development of our unique gene correction approach that we believe could offer a definitive cure for sickle cell patients,” said Josh Lehrer, M.D., M.Phil., chief executive officer of Graphite Bio. “This designation has the potential to accelerate the development of GPH101, which we are advancing with the goal of precisely and efficiently correcting the genetic mutation that is the underlying cause of sickle cell disease. We continue to enroll patients in our Phase 1/2 CEDAR trial and expect to dose our first patient later this year, with initial proof-of-concept data anticipated next year.”

The FDA’s Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. GPH101 was previously granted orphan drug designation by the FDA.

About GPH101 for Sickle Cell Disease
GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD). SCD is a serious, life-threatening inherited blood disorder that affects approximately 100,000 people in the United States and millions of people around the world, making it the most prevalent monogenic disease worldwide. GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle hemoglobin (HbS) production and restore adult hemoglobin (HbA) expression, thereby potentially curing SCD.

Click here to read the full article at BusinessWire

U.S. FDA Approves Supplemental New Drug Application for Expanded Indication of Oxbryta® for Children as Young as 4 Years of Age with Sickle Cell Disease

December 22, 2021

by administrator BPG Blog

Global Blood Therapeutics , Inc. (GBT) (NASDAQ: GBT) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval of a supplemental New Drug Application (sNDA) for Oxbryta^® (voxelotor) tablets for the treatment of sickle cell disease (SCD) in children ages 4 to less than 12 years. This approval expands the previously approved use of Oxbryta to treat SCD in patients ages 12 years and older in the United States. The FDA also approved GBT’s separate New Drug Application (NDA) for Oxbryta (voxelotor) tablets for oral suspension, a new dispersible, once-daily tablet dosage form suitable for patients ages 4 to less than 12 years as well as for older patients who have difficulty swallowing whole tablets. Oxbryta is the first and only approved medicine that directly targets sickle hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD.

“For decades, the sickle cell disease community has been profoundly underserved, and there have been limited treatment options for younger patients with their whole lives ahead of them. Complications of SCD that can cause irreversible organ damage are known to begin in the first few years of life, which is why earlier intervention is critical,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Today’s FDA approval of Oxbryta for children as young as 4 years old – and with a pediatric-friendly dosage form – is an important advance in the treatment of this devastating, lifelong condition. GBT is proud to lead the development of new medicines to address the inadequacies of care for SCD patients.”

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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