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

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

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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Neurocrine Biosciences Receives Orphan Drug Designation for Valbenazine as a Treatment for Chorea Associated with Huntington Disease

SAN DIEGO, May 12, 2022 /PRNewswire/ — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for valbenazine as a treatment for Huntington disease (HD). The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation. In December 2021, Neurocrine Biosciences reported top-line data from its Phase 3 KINECT-HD study evaluating the efficacy, safety and tolerability of valbenazine, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor being investigated as a once-daily treatment in adults with chorea associated with HD.

“Receiving an FDA Orphan Drug Designation validates our continued commitment to developing new treatment options that could benefit the lives of patients living with rare diseases, including those impacted by HD,” said Kevin Gorman, Ph.D., Chief Executive Officer. “We are in the process of completing data analysis from the KINECT-HD and the ongoing KINECT-HD2 studies, which will form the basis of our supplemental new drug application (sNDA) for submission to the FDA later this year.”

Enrollment is ongoing in the KINECT-HD2 open-label study to evaluate the long-term safety and tolerability of valbenazine for the treatment of chorea in Huntington Disease.

Click here to read the full article at Cision

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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Stealth Clinches Orphan Drug Designation in DMD

Stealth BioTherapeutics has received Orphan Drug Designation from the U.S. Food and Drug Administration for elamipretide. The candidate received this designation for its potential to treat patients with Duchenne Muscular Dystrophy (DMD), a rare neurologic disease that affects muscles. In addition, the FDA’s Division of Neurology has agreed to a pre-IND meeting to evaluate the path to regulatory approval of elamipretide.

DMD is typically diagnosed primarily in males between the ages of two and eleven years old, according to the National Institutes of Health’s Genetic and Rare Disease Information Center. Early signs of DMD include difficulty sitting, standing, walking and learning how to speak. As the disease progresses, muscles begin to weaken and degrade, leading to atrophy in skeletal and heart muscles. Patients often die in early adulthood, once respiratory or cardiac issues become too severe for treatment. The disease is hereditary, passed through an X-linked recessive chromosome pattern.

To combat the progression of DMD, elamipretide’s peptide compound composition allows it to penetrate the cellular membrane to bind to cardiolipin. After binding, elamipretide induces an increase in mitochondrial respiration and ATP production. Increased ATP production interrupts and potentially reverses oxidative stress. Oxidative stress is a factor in DMD, along with various other dry age-related macular degeneration and primary mitochondrial diseases.

Click here to read the full article at BioSpace

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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Can FDA replicate Operation Warp Speed for rare diseases? Not yet, but Peter Marks has some ideas

As COVID-19 continues to dominate the FDA’s bandwidth, Peter Marks, M.D., Ph.D., director of the agency’s Center for Biologics Evaluation and Research (CBER), is playing financial whack-a-mole to figure out where resources should flow. Topping his priority list is more money to regulate the budding cell and gene therapy field, an effort he hopes can at least partly mirror the regulator’s response to the pandemic. 

Marks’ comments came Tuesday on a call with the Alliance for a Stronger FDA, a lobbying group that advocates on behalf of the agency and its regulatory efforts, to discuss CBER’s budget priorities. Marks touched on a number of topics ranging from the need to bolster the agency’s overall IT infrastructure to improving staff retention. But most notable were his calls to speed up the center’s cell and gene therapy review efforts, which he said have failed to meet his expectations on account of the workload. 

“We are clearly in a position that we are not giving the kind of feedback that I’m comfortable with in real-time … to both vaccine developers, and particularly for those in the gene and cell therapy field,” he said. 

Click here to read the full article at Fierce Biotech

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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Belite Bio Receives FDA Fast Track Designation For LBS-008

SAN DIEGO, May 03, 2022 (GLOBE NEWSWIRE) — Belite Bio, Inc (the “Company”) (Nasdaq: BLTE),  a San Diego based clinical stage biopharmaceutical drug development company targeting currently untreatable eye diseases, such as atrophic Age-related Macular Degeneration (dry AMD) and Stargardt disease (STGD1), and metabolic diseases, today announced that LBS-008, an orally administered tablet, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of STGD1. This decision was based upon FDA’s review of non-clinical data and preliminary clinical data from studies of LBS-008.

“We are delighted that LBS-008 has received FDA Fast Track Designation. STGD1 is a terrible retinal disease with the potential to severely affect the vision of afflicted patients and there are currently no approved treatments,” said Dr. Tom Lin, the Company’s Chairman and CEO. “At present, we are conducting a Phase 3 clinical trial in order to bring to market a treatment that will halt or slow the progression of STGD1. Additionally, we are evaluating our plan to launch a Phase 2/3 trial in Dry AMD in 2022. Dry AMD is a disease which shares a similar underlying pathophysiology with STGD1 and is a leading cause of central vision loss in people over 50.”

The Company expects the next near-term data readout in its STGD1 Phase 2 trial to occur in the last quarter of this year when all subjects have completed 12 months of treatment.

Click here to read the full article at GlobeNewswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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Pliant Therapeutics Receives FDA Fast Track Designation for PLN-74809 for the Treatment of Idiopathic Pulmonary Fibrosis

SOUTH SAN FRANCISCO, Calif., May 03, 2022 (GLOBE NEWSWIRE) — Pliant Therapeutics (NASDAQ: PLRX) announced today that PLN-74809, its oral, dual-selective αvß6vß1 integrin inhibitor, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the potential treatment of idiopathic pulmonary fibrosis (IPF). PLN-74809, the Company’s lead drug candidate, is currently being tested as part of the INTEGRIS-IPF Phase 2a clinical trial (NCT04396756). Pliant anticipates topline data from this randomized, double-blind, placebo-controlled trial in patients with IPF, in mid-2022.

“The Fast Track designation marks an important step in PLN-74809’s clinical development in IPF. It underscores the urgent need for new therapeutic options to address this devastating disease,” said Éric Lefebvre, M.D., Chief Medical Officer at Pliant Therapeutics. “We will continue to work closely with the FDA to support the future development of PLN-74809 beginning with data from the Phase 2a INTEGRIS-IPF trial, which is on track for readout in mid-2022.”

FDA’s Fast Track designation is intended to facilitate and expedite the development and review of new drugs to treat serious or life-threatening conditions. To qualify, available clinical and non-clinical data need to demonstrate the potential to address unmet medical need. The benefits of Fast Track designation include opportunities for frequent meetings with the FDA to discuss trial design, development plans and data needed to support drug approval, as well as the ability to submit a New Drug Application (NDA) on a rolling basis, and eligibility for priority review, if relevant criteria are met.

Click here to read the full article at GlobeNewswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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

“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

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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Orphalan Touts First FDA-Approved Wilson’s Disease Treatment Since 1970

Orphalan announced the U.S. Food and Drug Administration has granted full approval to Cuvrior (trientine tetrahydrochloride) for the treatment of Wilson’s Disease, a rare genetic disorder. The approval announcement marks the first new treatment available for patients in over five decades.

In patients with Wilson’s Disease, the body is prevented from removing extra copper, which causes the element to build up in the liver, brain, eyes and other organs. The disease is caused by the disruption of or mutations to the ATP7B gene, which plays an important role in the movement of excess copper from the liver to the bile that is eventually excreted from the body. Approximately one in 90 people may be carriers of the disease gene, and the disease is estimated to impact 9,000 people in the U.S.

Liver dysfunction is one of the hallmarks of the disease, and typical symptoms of liver dysfunction, like swelling of the legs and abdomen and jaundice, are among the first signs of Wilson’s Disease. One of the tell-tale signs of the disease is a Kayser-Fleischer ring, a brownish-yellow ring in the eye caused by excess copper on the inner surface of the cornea.

Click here to read the full article at BioSpace

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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IDEAYA Biosciences Receives Orphan Drug Designation for Darovasertib, a PKC Inhibitor, for the Treatment of Uveal Melanoma

SOUTH SAN FRANCISCO, Calif., May 2, 2022 /PRNewswire/ — IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a synthetic lethality focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to darovasertib, a potential first-in-class protein kinase C (PKC) inhibitor, for the treatment of uveal melanoma.

IDEAYA is currently evaluating the synthetic lethal combination of darovasertib, a PKC inhibitor, and crizotinib, a cMET inhibitor, in patients with metastatic uveal melanoma (MUM) and in patients with GNAQ or GNA11 mutant solid tumors, in an ongoing Phase 1/2 clinical trial (NCT03947385) pursuant to a clinical trial collaboration and supply agreement with Pfizer. 

“We are excited to advance darovasertib towards a potential registration-enabling trial in metastatic uveal melanoma, and the orphan-drug designation is an important step towards our goal to bring this novel therapy to patients,” said Matthew Maurer, M.D., Vice President, Head of Clinical Oncology and Medical Affairs, at IDEAYA Biosciences.

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.

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SQZ Biotechnologies Receives FDA Fast Track Designation for its Lead Cell Therapy Candidate for the Treatment of HPV16+ Tumors

WATERTOWN, Mass.–(BUSINESS WIRE)–SQZ Biotechnologies (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the company’s lead cell therapy candidate, SQZ-PBMC-HPV, for the treatment of HPV16+ advanced or metastatic solid tumors.

Fast Track Designation is designed to accelerate the development and review of treatments for serious and life-threatening diseases where no treatment currently exists or where the treatment in discovery may be better than what is currently available.

“We are thrilled to receive FDA Fast Track Designation for our SQZ® Antigen Presenting Cells product candidate,” said Armon Sharei, Ph.D., Chief Executive Officer and Founder at SQZ Biotechnologies. “This designation adds to our exciting clinical data presented at ESMO-IO last year where we first demonstrated the potential of our Cell Squeeze® technology to drive clinical benefit while maintaining favorable tolerability. The FDA Fast Track program can potentially expedite future review processes and accelerate the registrational path for SQZ-PBMC-HPV.”

Click here to read the full article at BusinessWire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

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.