gene therapy Archives - BioPharma Global

PTC’s first gene therapy gains European regulator’s backing

June 15, 2022

The recommendation from the EMA’s human drugs committee is good news for the gene therapy field after a string of negative developments. Clinical and regulatory setbacks have weighed on company valuations, limiting their fundraising options and leading to restructuring and layoffs for a number of gene therapy developers.

PTC’s regulatory decision is the first of several expected in the coming months that, if positive, could help turn investors’ views around. After its European disappointment, Bluebird is expecting Food and Drug Administration decisions on its two gene therapies this year, while closely watched projects from BioMarin Pharmaceutical and UniQure could also be under FDA review soon. Likewise, PTC expects by the end of September to ask the FDA for approval of Upstaza.

PTC’s therapy corrects a mutation that disrupts production of an enzyme called aromatic L-amino acid decarboxylase, which is in turn vital to production of other chemicals needed for nervous system signaling. As with Luxturna and Zolgensma, Upstaza uses an adeno-associated virus to deliver the corrected gene into cells.

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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.

ASC Therapeutics Receives Key Regulatory Designations in U.S. and Europe to Advance its Second-Generation Gene Therapy for Hemophilia A

March 30, 2022

by administrator BPG Blog

ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic, metabolic, and other rare diseases has received from the United States (U.S.) Food and Drug Administration (FDA) the Fast Track Designation for ASC618, a second-generation gene therapy of hemophilia A. In addition, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion for an Orphan Medical Product Designation of ASC618.

In conjunction with the U.S. FDA IND clearance in 2021 and U.S. FDA Orphan Drug Designation in 2020 for ASC618, these regulatory achievements will significantly facilitate the scientific and clinical development of ASC 618’s gene replacement therapies for hemophilia A, reflecting:

A novel second-generation approach to achieve a safe, durable, and stable transgene expression to overcome the challenges of factor VIII replacement therapies.
The viral construct’s critical role with decreasing the therapeutic dose and minimizing cellular stress by increasing synthesis and secretion of factor VIII.

Ruhong Jiang, PhD, Chief Executive Officer at ASC Therapeutics, stated, “We are very pleased with the FDA’s and EMA’s regulatory decisions regarding ASC618. This brings us one step closer for providing a truly novel therapeutic approach for hemophilia A, providing potentially a functional cure for patients who currently require life-long care.”

Oscar Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added, “These regulatory milestones culminate years of hard work, dedication, and collaborative efforts by our functional teams. Pre-clinical studies have shown that ASC618 has the potential to reduce therapeutic dosing and increase durability of hemophilia A gene therapy with a novel bioengineered construct that can improve biosynthesis, protein folding, and secretion of factor VIII.”

Click here to read the full article at Businesswire

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Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

February 28, 2022

by administrator BPG Blog

Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today recognizes International Rare Disease Day by reflecting on successes and highlighting challenges that remain in bringing hope to 300 million patients worldwide who suffer from more than 7,000 rare diseases.

“As a world leader in gene therapy, Genethon is making a unique contribution to finding treatments and cures for rare diseases,” said Frederic Revah, Ph.D., Genethon CEO. “Products emerging from our research make up a global pipeline of treatments for patients who have no therapeutic alternatives. Today, thousands of patients around the world are already benefiting from our research.”

Products from Genethon’s research include a marketed gene therapy for spinal muscular atrophy and 12 other treatments in clinical trials for rare diseases of the muscle, immune system, eye, and liver. Another seven products from Genethon’s research should enter clinic trials in the next few years.

However, Dr. Revah observes. “While therapeutic successes are multiplying, demonstrating the full relevance of this breakthrough technology, the development of gene therapy for rare diseases is coming up against scientific, technological, clinical, economic and financial obstacles, which jeopardize making it available to as many people as possible.”

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Astellas Gene Therapy for Pompe Disease Clears Important Safety Hurdle

February 11, 2022

by administrator BPG Blog

Preliminary data from Astellas Pharma’s gene therapy study in adult patients with Pompe disease has crossed a first safety hurdle. At WORLDSymposium, Astellas presented interim safety data from a Phase I/II study assessing its AAV gene therapy for the rare, autosomal metabolic disease.

Weston Miller, M.D., senior medical director of clinical development at Astellas Gene Therapies, called the positive interim and tolerability data an important waypoint in the development of AT845, the company’s investigational adeno-associated virus (AAV) gene replacement therapy.

AT845 has been designed to deliver a functional alpha-glucosidase (GAA) gene into a patient in order to express acid alpha-glucosidase (GAA) directly into the muscle cells of adults with Late-Onset Pompe Disease (LOPD). GAA is responsible for metabolizing glycogen. The absence of GAA or its improper functioning causes the excessive accumulation of glycogen, primarily in the skeletal and cardiac muscles. Once it has accumulated in significant amounts, the glycogen causes damage to tissue structure and function, Miller said. AT845 is being assessed in the global Phase I/II FORTIS first-in-human clinical study.

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bluebird bio Announces FDA Priority Review of Biologics License Application for Gene Therapy for Cerebral Adrenoleukodystrophy

December 27, 2021

by administrator BPG Blog

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for elivaldogene autotemcel (eli-cel, Lenti-D®), the company’s gene therapy for cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age. Eli-cel is an investigational one-time gene therapy, custom-designed to treat the underlying cause of this irreversible neurodegenerative disease and to stabilize neurologic function. The agency set a Prescription Drug User Fee Act (PDUFA) goal date of June 17, 2022.

“Eli-cel is an important potential therapeutic option for patients with CALD—a devastating neurodegenerative disease—and we are encouraged to be moving forward given the urgent unmet need for these children and their families,” said Andrew Obenshain, chief executive officer, bluebird bio. “As the second BLA acceptance for bluebird bio this year, this is a meaningful milestone in our work to deliver one-time treatments for severe genetic diseases.”

If approved, eli-cel will be the first approved treatment to address the underlying genetic cause of disease for patients living with CALD in the U.S. – offering appropriate patients an alternative to allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with serious potential complications and mortality that increase in patients without a matched sibling donor. It is estimated that more than 70% of patients diagnosed with CALD do not have a matched sibling donor.

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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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Longeveron Granted Orphan Drug Designation by FDA for Lomecel-B to Treat Infants with Hypoplastic Left Heart Syndrome (HLHS)

December 7, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Longeveron Inc. (NASDAQ: LGVN) (“Longeveron” or “Company”), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

“Adding to the Rare Pediatric Disease (RPD) designation already granted to Lomecel-B for treatment of HLHS, the FDA’s decision to grant ODD to Lomecel-B for this indication indicates the ongoing and unmet need for new therapies to treat infants with HLHS,” said Geoff Green, Chief Executive Officer at Longeveron. “Building on results from our completed Phase 1 safety-focused trial, we believe Lomecel-B has potential to improve outcomes for these severely impacted infants by way of repairing cardiac tissue and improving ventricular function. The combination of both RPD and ODD allows us to potentially move more efficiently through clinical development and regulatory review, and Lomecel-B may be eligible for a period of marketing exclusivity upon approval for this indication.”

To Read the Complete Article at BioSpace News Click Here

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Soligenix Granted Pediatric Investigational Plan Waiver for HyBryte™ in CTCL in the United Kingdom

December 6, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Soligenix (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has been granted a Pediatric Investigation Plan (PIP) product-specific waiver from the Medicines and Healthcare products Regulatory Agency (MHRA) for HyBryte™ (SGX301 or synthetic hypericin), which has successfully concluded a Phase 3 pivotal clinical study for the treatment of early stage cutaneous T-cell lymphoma (CTCL).

The waiver was provided for all subsets of the pediatric population from birth to less than 18 years of age on the grounds that clinical studies in this rare population are not feasible. Earlier this year the European Medicines Agency (EMA) also granted a waiver to the Pediatric Investigational Plan requirements for the European Union (EU). With the withdrawal of the United Kingdom (UK) from the EU effective January 1, 2021, the MHRA became the UK’s standalone medicines and medical devices regulator.

“This achievement is an important regulatory milestone as we move forward with marketing applications worldwide,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. “The PIP waiver allows us to work towards advancing a marketing authorization application (MAA) in the UK in a more cost-effective manner since we will not need to expend resources to conduct a pediatric clinical study.”

To Read the Complete Article at BioSpace News Click Here

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Reata Pharmaceuticals Receives Fast Track Designation From the FDA for Omaveloxolone for the Treatment of Friedreich’s Ataxia

December 3, 2021

by administrator Drug Discovery News Rare Disease Highlight Rare Disease News Regulatory News

Reata Pharmaceuticals, Inc. (Nasdaq: RETA), (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, today announced the U.S. Food and Drug Administration (“FDA”) has granted Fast Track Designation for omaveloxolone for the treatment of Friedreich’s ataxia.

“We are pleased to receive Fast Track Designation as it highlights the potential of omaveloxolone to address a significant unmet medical need for the treatment of patients with Friedreich’s ataxia, a severe and devastating disease,” said Warren Huff, Reata’s President and Chief Executive Officer. “We remain committed to submitting our New Drug Application during the first quarter of 2022 and continue working with the FDA to secure regulatory approval as quickly as possible.”

The Fast Track program is designed to accelerate the development and review of products such as omaveloxolone, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track Designation enables more frequent communication with the FDA and eligibility for FDA programs such as priority review and rolling review, if relevant criteria are met.

About Friedreich’s Ataxia

Friedreich’s ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder, which is normally diagnosed during adolescence. Friedreich’s ataxia is caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin. Pathogenic repeat expansions can lead to impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production. Patients with Friedreich’s ataxia experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their teens or early 20s. Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy. Based on literature and proprietary research, we believe Friedreich’s ataxia affects approximately 5,000 children and adults in the United States and 22,000 individuals globally. There are currently no approved therapies for the treatment of patients with Friedreich’s ataxia.

To Read the Complete Article at BioSpace News Click Here

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U.S. FDA Grants Bionomics Fast Track Designation to BNC210 for the Acute Treatment of Social Anxiety Disorder and Other Anxiety Related Disorders

December 2, 2021

by administrator Drug Discovery News Regulatory News

Bionomics Limited (ASX:BNO, OTCQB:BNOEF) (Bionomics or Company), a clinical-stage biopharmaceutical company, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the BNC210 development program for the acute treatment of Social Anxiety Disorder (SAD) and other anxiety-related disorders. In November 2019, the FDA granted Fast Track designation to the BNC210 development program for the treatment of Post-Traumatic Stress Disorder (PTSD) and other trauma-related and stressor-related disorders.

Fast Track designation is a FDA program intended to facilitate and expedite development and review of new drugs that demonstrate the potential to address unmet medical need in the treatment of a serious or life-threatening disease or condition. A drug that receives Fast Track designation is eligible for some, or all, of the following:

more frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval;
more frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers;
eligibility for priority review and accelerated approval, if relevant criteria are met; and
possible review of the New Drug Application (NDA) on a rolling basis. NDA review usually does not begin until a company has submitted the entire drug application to the FDA. When an NDA is eligible for rolling review, FDA begins reviewing completed sections of an NDA before the entire NDA is submitted.

BNC210 is an oral proprietary selective negative allosteric modulator of the α7 nicotinic acetylcholine receptor in development for the acute treatment of SAD and chronic treatment of PTSD. Following encouraging results in a previous Phase 2a study in Generalised Anxiety Disorder (GAD) patients where a single oral dose administration of BNC210 showed significantly reduced threat-avoidance behaviour and significantly reduced connectivity between the amygdala and the anterior cingulate cortex, a network involved in regulating anxious responses to aversive stimuli, BNC210 will be evaluated as an acute, or single-dose, treatment for patients with SAD in a planned Phase 2 clinical trial named the PREVAIL Study that we expect to initiate by the end of 2021.

To Read the Complete Article at BioSpace News Click Here

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FDA Accepts Regulatory Submission of Supplemental New Drug Application for LYNPARZA® (olaparib) as Adjuvant Treatment in BRCA-Mutated, HER2-Negative High-Risk Early Breast Cancer and Grants Priority Review

December 1, 2021

by administrator Drug Discovery News Regulatory News

AstraZeneca and Merck & Co. (NYSE: MRK), known as MSD outside the United States and Canada, today announced that a supplemental New Drug Application (sNDA) for LYNPARZAhas been accepted and granted priority review by the U.S. Food and Drug Administration (FDA) for the adjuvant treatment of patients with BRCA-mutated (BRCAm), human epidermal growth factor receptor 2 (HER2)-negative high-risk early breast cancer who have already been treated with chemotherapy either before or after surgery. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date during the first quarter of 2022.

Breast cancer is now the most diagnosed cancer worldwide, with an estimated 2.3 million patients diagnosed in 2020. Nearly 91% of all breast cancer patients are diagnosed at an early stage of disease, and germline BRCA mutations are found in approximately 5% of patients.

The sNDA was based on results from the Phase 3 OlympiA trial presented during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and simultaneously published in The New England Journal of Medicine.

These results showedLYNPARZA demonstrated a statistically significant and clinically meaningful improvement in invasive disease-free survival (iDFS), reducing the risk of invasive breast cancer recurrence, second cancers or death by 42% versus placebo (HR=0.58 [99.5% CI, 0.41-0.82]; p<0.0001).

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