EMA Archives - BioPharma Global

ALX Oncology’s Evorpacept Receives Fast Track Designation from FDA as First-Line Treatment for Head and Neck Squamous Cell Carcinoma

August 22, 2022

SOUTH SAN FRANCISCO, Calif., Aug. 01, 2022 (GLOBE NEWSWIRE) — ALX Oncology Holdings Inc., (“ALX Oncology”) (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Fast Track designation to evorpacept, a next generation CD47 blocker, in combination with KEYTRUDA® (pembrolizumab), Merck’s anti-PD-1 therapy, for the first-line treatment of adult patients with PD-L1 positive advanced head and neck squamous cell carcinoma (“HNSCC”).

The FDA’s decision is informed by the results of ALX Oncology’s phase 1 clinical trial, ASPEN-01, that showed preliminary antitumor activity and a favorable safety profile in checkpoint inhibitor-naïve patients with second-line or greater advanced HNSCC treated with evorpacept and pembrolizumab (n=10). The preliminary objective response rate of 40% in this limited population represents an improvement over historical anti-PD-1 monotherapy activity in a similar population and supported the initiation of the ASPEN-03 (NCT04675294) study in May 2021 in collaboration with Merck (known as MSD outside the US and Canada). ASPEN-03 is a randomized phase 2 multi-center study to investigate the anti-tumor efficacy of evorpacept plus pembrolizumab in patients with first-line metastatic or unresectable, recurrent PD-L1 positive HNSCC.

“The FDA’s Fast Track designation for the first-line treatment of HNSCC with evorpacept in combination with pembrolizumab builds upon evorpacept’s prior Fast Track designation in the first-line HNSCC population in combination with pembrolizumab and standard chemotherapy highlighting the potential clinical utility of evorpacept in this difficult-to-treat disease,” said Sophia Randolph, M.D., Ph.D., Chief Medical Officer, ALX Oncology. “We are pleased by the patient enrollment progress being made across our HNSCC phase 2 program (NCT04675294; NCT04675333) as we seek to advance evorpacept to help patients living with this disease.”

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.

FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS

August 19, 2022

by administrator BPG Blog

CAMBRIDGE, Mass., July 26, 2022 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The application has been granted priority review and given a Prescription Drug User Fee Act action date of January 25, 2023. The FDA has noted that it is currently planning to hold an Advisory Committee meeting for this application, on a yet-to-be determined date. The average life expectancy for people with ALS is three to five years from time of symptom onset. There is currently no treatment targeted for SOD1-ALS.²

“The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS,” said Priya Singhal, M.D., M.P.H., Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen. “Pursuing the FDA’s accelerated approval pathway offers the potential to make tofersen available to people living with this fatal, neurodegenerative disease as quickly as possible. If approved, tofersen will be the first treatment to target a genetic cause of ALS and we hope this will pave the way for further advances in this relentless disease.”

Biogen is seeking approval of tofersen under the FDA’s accelerated approval pathway, based on the use of neurofilament as a surrogate biomarker that is reasonably likely to predict clinical benefit. Neurofilaments are normal proteins found in healthy neurons, that are increased in blood and cerebrospinal fluid when damage has been done to neurons or their axons and are a marker of neurodegeneration. In ALS, higher levels of neurofilaments have been found to predict more rapid decline in clinical function and shortened survival.³ Tofersen study results suggest reductions in neurofilament preceded and predicted slowing of decline in measures of clinical and respiratory function, strength, and quality of life. Biogen is committed to ongoing data generation and finalizing the confirmatory data package with the FDA.

Click here to read the full article at Biogen

FDA, in another test of its flexibility, agrees to review Biogen’s closely watched ALS drug

August 17, 2022

by administrator BPG Blog

Late last year, Biogen disclosed results from a closely watched study evaluating an experimental drug for Lou Gehrig’s disease, also known as ALS or amyotrophic lateral sclerosis. They showed the drug, called tofersen, wasn’t better than a placebo at slowing progression of the fatal nerve disorder, dealing yet another blow to patients and caregivers, who currently have very limited treatment options.

Despite those results, Biogen recently submitted an approval application to the Food and Drug Administration and on Tuesday, the company announced the agency has agreed to review it on an expedited basis. Biogen had not previously disclosed its submission.

A verdict on approval is expected by Jan. 25, the company said.

The FDA’s decision to review comes a little more than a year after it granted approval to another Biogen drug with a mixed track record in clinical testing. Last June, the drug, which is now sold as Aduhelm, became the first in the U.S. cleared to treat Alzheimer’s disease itself rather than its associated symptoms. While historic, the approval was also exceedingly controversial, and Biogen has since all but scrapped Aduhelm as a product because of challenges getting doctors to prescribe it and insurers to pay for it.

Click here to read the full article at Biopharma Dive

Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS

August 15, 2022

by administrator BPG Blog

CARLSBAD, Calif., July 26, 2022 /PRNewswire/ — Ionis Pharmaceuticals (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational antisense medicine for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS). The NDA was submitted by Biogen, which licensed tofersen from Ionis in 2018. The application has been granted priority review and given a Prescription Drug User Fee Act action date of Jan. 25, 2023. The FDA has noted that it is currently planning to hold an Advisory Committee meeting for this application. The average life expectancy for people with ALS is three to five years from time of symptom onset; patients with some SOD1 mutations have an even shorter life expectancy. There is currently no treatment targeted for SOD1-ALS.

“Acceptance of the new drug application for tofersen is a monumental milestone, not just for Ionis but for all people with SOD1-ALS, their families and healthcare professionals battling this devastating disease. To them we extend our deepest gratitude. Their courage has been instrumental to the achievement of this goal,” said C. Frank Bennett, Ph.D., executive vice president, chief scientific officer and franchise leader for neurological programs at Ionis. “We also want to thank Biogen for their commitment to advancing tofersen, which, if approved, will be the first treatment that targets a genetic cause of ALS.” Dr. Bennett added, “Acceptance of the NDA for tofersen further strengthens Ionis’ platform strategy to target all forms of ALS and central nervous system disorders more broadly.”

Biogen is seeking approval of tofersen under the FDA’s accelerated approval pathway, based on the use of neurofilament as a surrogate biomarker that is reasonably likely to predict clinical benefit. Neurofilaments are normal proteins found in healthy neurons, that are increased in blood and cerebrospinal fluid when damage has been done to neurons or their axons and are a marker of neurodegeneration. In ALS, higher levels of neurofilaments have been found to predict more rapid decline in clinical function and shortened survival. Tofersen study results suggest reductions in neurofilament preceded and predicted slowing of decline in measures of clinical and respiratory function, strength and quality of life. Biogen has stated its commitment to ongoing data generation and finalizing the confirmatory package with the FDA.

Click here to read the full article at Ionis

Pliant Therapeutics Receives FDA Fast Track Designation for PLN-74809 for the Treatment of Primary Sclerosing Cholangitis

August 12, 2022

by administrator BPG Blog

SOUTH SAN FRANCISCO, Calif., July 21, 2022 (GLOBE NEWSWIRE) — Pliant Therapeutics (NASDAQ: PLRX) announced today that PLN-74809, its oral, dual-selective α_vß₆/ α_vß₁integrin inhibitor, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the potential treatment of primary sclerosing cholangitis (PSC). PLN-74809, the Company’s lead drug candidate, is currently being tested as part of the INTEGRIS-PSC Phase 2a clinical trial (NCT04480840). Pliant anticipates topline data from this randomized, double-blind, placebo-controlled trial in patients with PSC, in the first half of 2023.

“Fast Track designation in primary sclerosing cholangitis represents a significant milestone in PLN-74809’s development,” said Éric Lefebvre, M.D., Chief Medical Officer at Pliant Therapeutics. “PSC is a deadly disease with no FDA-approved Therapies. Fast Track designation underscores the urgent need for new therapeutic options to address PSC and other fibrotic diseases.”

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

Wugen Receives U.S. FDA Fast Track and Rare Pediatric Disease Designations for WU-CART-007 for the Treatment of R/R T-ALL/LBL

August 10, 2022

by administrator BPG Blog

ST. LOUIS & SAN DIEGO–(BUSINESS WIRE)–Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation and Rare Pediatric Disease Designation (RPDD) for WU-CART-007, the company’s off-the-shelf CAR-T cell therapy. WU-CART-007 is currently being explored for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL).

“We are very pleased to have received both fast track and rare pediatric disease designations, which re-affirm the great unmet need for new treatment options for people with R/R T-ALL/LBL,” said Dan Kemp, Ph.D., President and Chief Executive Officer of Wugen. “Earlier this year, we dosed the first patient in our ongoing Phase 1/2 trial of WU-CART-007 for R/R T-ALL/LBL and are currently in the dose escalation phase of the study. We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development.”

The FDA’s Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, the purpose is to get important new drugs to the patient earlier. Features of Fast Track Designation include opportunities for more frequent interactions with the FDA review team and, if supported by clinical data, eligibility for Priority Review.

Click here to read the full article at Cision PR Newswire

AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome

August 8, 2022

by administrator BPG Blog

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AVR-RD-05, its gene therapy for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys.

Orphan drug designation is granted by FDA to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.

Hunter syndrome is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules. The gene therapy uses a patient’s own hematopoietic stem cells (HSCs) that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. The company’s planned collaborator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in 2023 under the company’s collaboration with the University of Manchester, UK. The program was developed by Brian Bigger, Ph.D., professor of cell and gene therapy at the University of Manchester, who has previously published preclinical data demonstrating that HSC gene therapy deploying an optimized, proprietary tag has the potential to correct peripheral disease and normalize brain pathology.

Click here to read the full article at Cision PR Newswire

Anthos Therapeutics Announces that Abelacimab has Received FDA Fast Track Designation for the Treatment of Thrombosis Associated with Cancer

August 5, 2022

by administrator BPG Blog

CAMBRIDGE, Mass., July 11, 2022 /PRNewswire/ — Anthos Therapeutics, a clinical-stage biotechnology company developing innovative therapies for cardiovascular and metabolic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its investigational Factor XI inhibitor, abelacimab, for the treatment of thrombosis associated with cancer. The company will also be announcing this important milestone today at a session of the ongoing 2022 Congress of the International Society on Thrombosis and Haemostasis (ISTH) Congress in London, UK.

The Fast Track Designation process is designed to facilitate the development and expedite the review of treatments for serious medical conditions, thereby, addressing unmet medical needs. Drugs that are included in this program may be eligible for more frequent interactions with the FDA to discuss the development path, and if the program criteria are met, eligibility for a potential Rolling Review, Accelerated Approval, and Priority Review.

Venous Thromboembolism (VTE), including both deep vein thrombosis and pulmonary embolism, is the second most prevalent cause of death in patients with cancer, second only to the disease itself.¹ However, treatment of Cancer Associated Thrombosis (CAT) can be challenging because the currently available anticoagulants used to treat VTE can have an increased risk of bleeding.^2,3

Click here to read the full article at Cision PR Newswire

Phanes Therapeutics’ PT886 granted Orphan Drug Designation for the treatment of pancreatic cancer by the FDA

August 3, 2022

by administrator BPG Blog

SAN DIEGO, June 30, 2022 /PRNewswire/ — Phanes Therapeutics, Inc. (Phanes), an emerging leader in innovative discovery research and clinical development in oncology, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT886 for the treatment of pancreatic cancer. Earlier this month, the FDA granted orphan drug designation to the company’s PT217 for the treatment of small cell lung cancer.

PT886 is a first-in-class bispecific antibody targeting claudin 18.2 (CLDN18.2) and cluster of differentiation 47 (CD47) being developed for patients with pancreatic cancer as well as gastric and gastroesophageal cancers. Pancreatic cancer is an aggressive form of cancer characterized by high mortality rates and significant morbidities. The 5-year survival rate of patients with pancreatic cancer is only 10.8% and projections for 2022 estimate that approximately 50,000 Americans will die of pancreatic cancer this year and by 2030 is projected to exceed breast, prostate, and colorectal malignancies as the leading cause of cancer-related deaths in the US.

“PT886 has the potential to be a transformative treatment option for pancreatic cancer patients whose current standard of care is severely limited.” said Dr. Ming Wang, Founder and CEO of Phanes Therapeutics. “This orphan drug designation comes in the same month with our recent IND clearance for PT886, which we are rapidly progressing into the clinic. These important milestones the company has achieved in Q2 this year follow the March IND clearance for PT199, an anti-CD73 monoclonal antibody for the treatment of multiple solid tumors.”

Click here to read the full article at Cision PR Newswire

Aura Biosciences Receives FDA Fast Track Designation for Belzupacap Sarotalocan (AU-011) for the Treatment of Non-Muscle Invasive Bladder Cancer

August 1, 2022

by administrator BPG Blog

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for belzupacap sarotalocan (AU-011), Aura’s first VDC product candidate, for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC).

“We are pleased that belzupacap sarotalocan has been granted Fast Track designation. We believe that, given that NMIBC presents such a high unmet medical need, the opportunity for more frequent interactions with Division of Oncology at FDA and the potential for Priority Review will be valuable as we advance further into clinical development in patients with NMIBC,” said Dr. Mark De Rosch, Chief Operating Officer and Head of Regulatory Affairs of Aura.

“NMIBC has no approved targeted therapies, and patients experience high levels of recurrence and progression, ultimately leading to cystectomy or the removal of the entire bladder,” said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura. “This milestone supports our goal to advance the development of belzupacap sarotalocan for patients with NMIBC in need of better and earlier targeted treatment options with the potential to help preserve the bladder.”

Click here to read the full article at BusinessWire