Notizia - Recent Pharma, Healthcare and Biotech Happenings
Aug 19, 2025
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Precigen, Inc. announced that the FDA has granted full approval to PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). The decision makes PAPZIMEOS the first and only FDA-approved therapy for RRP, a rare and debilitating disease caused by chronic HPV 6 or HPV 11 infection. Unlike previous accelerated approvals, the agency’s decision does not require a confirmatory trial, underscoring the strength of the pivotal data.
“For more than a century, patients with RRP have relied on repeated surgeries to manage this relentless condition. Today marks a historic turning point,” said Helen Sabzevari, PhD, President and CEO of Precigen. “With the landmark approval of PAPZIMEOS, adult patients now have access to the first therapy that targets the root cause of the disease.”
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RRP, which affects an estimated 27,000 adult patients in the U.S., often leads to severe voice disturbance, airway compromise, and recurrent infections. Current management has centered on repeated surgical removal of tumors, a burdensome approach that does not prevent recurrence. PAPZIMEOS, a non-replicating adenoviral vector-based immunotherapy, is administered through four subcutaneous injections over 12 weeks and is designed to elicit an immune response against papilloma cells expressing HPV 6/11 proteins.
“This long-awaited FDA approval represents a momentous milestone for the RRP community,” said Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation. “For the first time, adult patients with RRP have access to an FDA-approved therapy that offers the potential to reduce—or even eliminate—endless repeated surgeries.”
The approval was supported by data from a pivotal open-label, single-arm study in adults with RRP. Results showed that 51% of patients achieved a Complete Response, requiring no surgeries in the 12 months following treatment, with durability beyond 24 months in many cases. PAPZIMEOS was well-tolerated, with no dose-limiting toxicities or treatment-related adverse events greater than Grade 2.
Tonix Pharmaceuticals Holding Corp. announced that the FDA has approved Tonmya (cyclobenzaprine HCl sublingual tablets) for the treatment of fibromyalgia in adults. Tonmya, a first-in-class, non-opioid bedtime analgesic, is the first new therapy for fibromyalgia in more than 15 years, addressing a major unmet need for millions of patients in the U.S.
“The FDA approval of Tonmya as a first-line treatment represents a landmark advancement for people living with fibromyalgia,” said Seth Lederman, M.D., CEO of Tonix Pharmaceuticals. “By targeting nonrestorative sleep, a hallmark of this condition, Tonmya has the potential to provide meaningful relief and improve quality of life for patients.”
Fibromyalgia affects more than 10 million adults in the U.S., primarily women, and is marked by chronic pain, fatigue, and sleep disturbances. The FDA’s decision was supported by two pivotal Phase III randomized trials involving nearly 1,000 patients, where Tonmya significantly reduced daily pain scores versus placebo and demonstrated meaningful improvements in patient-reported outcomes. Across more than 1,400 patients evaluated in Phase III studies, the drug was generally well tolerated.
“For over 15 years, this community has been underserved and waiting for new treatment options,” said Sharon Waldrop, founder of the Fibromyalgia Association. “This approval is a promising step forward and brings renewed hope to millions who live with this painful and exhausting condition.”
Tonmya is expected to be available for U.S. adult patients with fibromyalgia beginning in the fourth quarter of this year.
REGENXBIO Inc. announced that the FDA has extended its review timeline for the Biologics License Application (BLA) of clemidsogene lanparvovec (RGX-121), an investigational one-time gene therapy for Mucopolysaccharidosis II (Hunter syndrome). The Prescription Drug User Fee Act (PDUFA) goal date has been moved from November 9, 2025, to February 8, 2026, following the submission of additional long-term clinical data.
“Boys with this rare, devastating disease have no treatment options to address neurodevelopmental decline, and the Hunter syndrome community is in urgent need of a therapeutic option with the potential to improve these patients’ lives,” said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. “We promptly provided the FDA with the information requested, and our commercial launch plans remain on track.”
The extension is based on the company’s submission of positive 12-month clinical results from all 13 patients in the pivotal study of RGX-121. These outcomes are consistent with earlier biomarker and neurodevelopmental data and will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in September 2025.
RGX-121 has already received multiple regulatory designations, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) status from the FDA, along with ATMP classification from the European Medicines Agency. No safety concerns have been raised during the FDA’s review or inspections to date.
SystImmune Inc. and Bristol Myers Squibb announced that the FDA has granted Breakthrough Therapy Designation (BTD) to izalontamab brengitecan (iza-bren) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR exon 19 deletions or exon 21 L858R mutations whose disease has progressed after EGFR tyrosine kinase inhibitors (TKIs) and platinum-based chemotherapy.
“The FDA’s granting of Breakthrough Therapy Designation underscores the potential of iza-bren to meaningfully improve clinical outcomes for patients with previously treated EGFR-mutant NSCLC,” said Dr. Jonathan Cheng, Chief Medical Officer of SystImmune. “The data we have generated to date suggest that iza-bren could address a critical unmet need, and we look forward to working closely with the FDA to advance development.”
Iza-bren is a potential first-in-class bispecific antibody-drug conjugate (ADC) targeting EGFR and HER3, carrying a topoisomerase I inhibitor payload. Developed by Biokin in China and jointly advanced by SystImmune and Bristol Myers Squibb outside China, the therapy aims to address the lack of effective options for patients whose disease progresses following frontline EGFR TKI therapy and platinum chemotherapy.
The FDA’s decision was supported by efficacy and safety results from three ongoing trials: BL-B01D1-101 and BL-B01D1-203 in China, and the global BL-B01D1-LUNG-101 study across the U.S., Europe, and Japan. Collectively, these studies demonstrated promising clinical activity and a manageable safety profile in heavily pretreated EGFR-mutant NSCLC patients.
Soligenix, Inc. announced that the FDA has granted orphan drug designation to dusquetide, the active ingredient in SGX945, for the treatment of Behçet’s Disease. The decision follows positive Phase IIa results demonstrating biological efficacy and safety in patients with oral ulcers caused by the rare autoimmune disorder.
“Behçet’s Disease is an area of unmet medical need, with as many as one million people worldwide affected by this incurable condition,” said Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. “Given the meaningful improvements seen in our proof-of-concept trial, we are hopeful dusquetide can play a role in helping underserved patients. The FDA’s orphan designation is an important milestone as we continue to advance SGX945.”
Under the U.S. Orphan Drug Act, therapies that receive orphan designation are eligible for incentives, including seven years of market exclusivity upon approval, tax credits, potential grant funding, and waiver of FDA user fees.
The Phase IIa study of SGX945 in Behçet’s Disease patients showed encouraging efficacy in treating oral aphthous ulcers, a hallmark symptom that contributes significantly to the disease burden. Soligenix plans to leverage orphan designation benefits to accelerate clinical development and strengthen its intellectual property position around dusquetide.
Article in PDF
Aug 19, 2025
Table of Contents
Precigen, Inc. announced that the FDA has granted full approval to PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). The decision makes PAPZIMEOS the first and only FDA-approved therapy for RRP, a rare and debilitating disease caused by chronic HPV 6 or HPV 11 infection. Unlike previous accelerated approvals, the agency’s decision does not require a confirmatory trial, underscoring the strength of the pivotal data.
“For more than a century, patients with RRP have relied on repeated surgeries to manage this relentless condition. Today marks a historic turning point,” said Helen Sabzevari, PhD, President and CEO of Precigen. “With the landmark approval of PAPZIMEOS, adult patients now have access to the first therapy that targets the root cause of the disease.”
RRP, which affects an estimated 27,000 adult patients in the U.S., often leads to severe voice disturbance, airway compromise, and recurrent infections. Current management has centered on repeated surgical removal of tumors, a burdensome approach that does not prevent recurrence. PAPZIMEOS, a non-replicating adenoviral vector-based immunotherapy, is administered through four subcutaneous injections over 12 weeks and is designed to elicit an immune response against papilloma cells expressing HPV 6/11 proteins.
“This long-awaited FDA approval represents a momentous milestone for the RRP community,” said Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation. “For the first time, adult patients with RRP have access to an FDA-approved therapy that offers the potential to reduce—or even eliminate—endless repeated surgeries.”
The approval was supported by data from a pivotal open-label, single-arm study in adults with RRP. Results showed that 51% of patients achieved a Complete Response, requiring no surgeries in the 12 months following treatment, with durability beyond 24 months in many cases. PAPZIMEOS was well-tolerated, with no dose-limiting toxicities or treatment-related adverse events greater than Grade 2.
Tonix Pharmaceuticals Holding Corp. announced that the FDA has approved Tonmya (cyclobenzaprine HCl sublingual tablets) for the treatment of fibromyalgia in adults. Tonmya, a first-in-class, non-opioid bedtime analgesic, is the first new therapy for fibromyalgia in more than 15 years, addressing a major unmet need for millions of patients in the U.S.
“The FDA approval of Tonmya as a first-line treatment represents a landmark advancement for people living with fibromyalgia,” said Seth Lederman, M.D., CEO of Tonix Pharmaceuticals. “By targeting nonrestorative sleep, a hallmark of this condition, Tonmya has the potential to provide meaningful relief and improve quality of life for patients.”
Fibromyalgia affects more than 10 million adults in the U.S., primarily women, and is marked by chronic pain, fatigue, and sleep disturbances. The FDA’s decision was supported by two pivotal Phase III randomized trials involving nearly 1,000 patients, where Tonmya significantly reduced daily pain scores versus placebo and demonstrated meaningful improvements in patient-reported outcomes. Across more than 1,400 patients evaluated in Phase III studies, the drug was generally well tolerated.
“For over 15 years, this community has been underserved and waiting for new treatment options,” said Sharon Waldrop, founder of the Fibromyalgia Association. “This approval is a promising step forward and brings renewed hope to millions who live with this painful and exhausting condition.”
Tonmya is expected to be available for U.S. adult patients with fibromyalgia beginning in the fourth quarter of this year.
REGENXBIO Inc. announced that the FDA has extended its review timeline for the Biologics License Application (BLA) of clemidsogene lanparvovec (RGX-121), an investigational one-time gene therapy for Mucopolysaccharidosis II (Hunter syndrome). The Prescription Drug User Fee Act (PDUFA) goal date has been moved from November 9, 2025, to February 8, 2026, following the submission of additional long-term clinical data.
“Boys with this rare, devastating disease have no treatment options to address neurodevelopmental decline, and the Hunter syndrome community is in urgent need of a therapeutic option with the potential to improve these patients’ lives,” said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. “We promptly provided the FDA with the information requested, and our commercial launch plans remain on track.”
The extension is based on the company’s submission of positive 12-month clinical results from all 13 patients in the pivotal study of RGX-121. These outcomes are consistent with earlier biomarker and neurodevelopmental data and will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in September 2025.
RGX-121 has already received multiple regulatory designations, including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) status from the FDA, along with ATMP classification from the European Medicines Agency. No safety concerns have been raised during the FDA’s review or inspections to date.
SystImmune Inc. and Bristol Myers Squibb announced that the FDA has granted Breakthrough Therapy Designation (BTD) to izalontamab brengitecan (iza-bren) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR exon 19 deletions or exon 21 L858R mutations whose disease has progressed after EGFR tyrosine kinase inhibitors (TKIs) and platinum-based chemotherapy.
“The FDA’s granting of Breakthrough Therapy Designation underscores the potential of iza-bren to meaningfully improve clinical outcomes for patients with previously treated EGFR-mutant NSCLC,” said Dr. Jonathan Cheng, Chief Medical Officer of SystImmune. “The data we have generated to date suggest that iza-bren could address a critical unmet need, and we look forward to working closely with the FDA to advance development.”
Iza-bren is a potential first-in-class bispecific antibody-drug conjugate (ADC) targeting EGFR and HER3, carrying a topoisomerase I inhibitor payload. Developed by Biokin in China and jointly advanced by SystImmune and Bristol Myers Squibb outside China, the therapy aims to address the lack of effective options for patients whose disease progresses following frontline EGFR TKI therapy and platinum chemotherapy.
The FDA’s decision was supported by efficacy and safety results from three ongoing trials: BL-B01D1-101 and BL-B01D1-203 in China, and the global BL-B01D1-LUNG-101 study across the U.S., Europe, and Japan. Collectively, these studies demonstrated promising clinical activity and a manageable safety profile in heavily pretreated EGFR-mutant NSCLC patients.
Soligenix, Inc. announced that the FDA has granted orphan drug designation to dusquetide, the active ingredient in SGX945, for the treatment of Behçet’s Disease. The decision follows positive Phase IIa results demonstrating biological efficacy and safety in patients with oral ulcers caused by the rare autoimmune disorder.
“Behçet’s Disease is an area of unmet medical need, with as many as one million people worldwide affected by this incurable condition,” said Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. “Given the meaningful improvements seen in our proof-of-concept trial, we are hopeful dusquetide can play a role in helping underserved patients. The FDA’s orphan designation is an important milestone as we continue to advance SGX945.”
Under the U.S. Orphan Drug Act, therapies that receive orphan designation are eligible for incentives, including seven years of market exclusivity upon approval, tax credits, potential grant funding, and waiver of FDA user fees.
The Phase IIa study of SGX945 in Behçet’s Disease patients showed encouraging efficacy in treating oral aphthous ulcers, a hallmark symptom that contributes significantly to the disease burden. Soligenix plans to leverage orphan designation benefits to accelerate clinical development and strengthen its intellectual property position around dusquetide.
