NeuroNOS Secures FDA Orphan Drug Designation for BA-101 in Glioblastoma

NeuroNOS, a biopharmaceutical company specializing in neurological disorders and neuro-oncology and a subsidiary of Beyond Air, announced that the FDA has granted Orphan Drug Designation (ODD) to its lead investigational therapy, BA-101, for the treatment of glioblastoma (GBM). GBM is an aggressive primary brain tumor with limited treatment options, and current standard-of-care therapies, surgery, radiation, and temozolomide, are not curative, with a median survival of under 12 months.

“We are pleased to receive orphan drug designation from the FDA for the treatment of glioblastoma,” said Amir Avniel, CEO of NeuroNOS. “This marks our entrance into oncology and underscores our mission to bring targeted therapies to individuals affected by rare neurological conditions. Emerging research shows that nitric oxide is a key modulator in GBM therapy response, and we believe our approach to balance NO levels could transform outcomes for patients.”

FDA ODD is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides incentives, including tax credits for clinical trials and user fee waivers, as well as seven years of market exclusivity upon approval, independent of intellectual property rights.

“Glioblastoma represents a profound unmet need,” said Prof. Haitham Amal, CSO of NeuroNOS. “Our published and unpublished data show a strong link between nitric oxide and GBM. We are committed to working closely with regulators, investigators, patient groups, and foundations to advance BA-101 toward first-in-human studies.”

FDA Expands VONVENDI Approval by Takeda for Von Willebrand Disease in Adults and Children

Takeda announced that the FDA has approved the supplemental Biologics License Application (sBLA) for VONVENDI [von Willebrand factor (Recombinant)], broadening its indication to include routine prophylaxis to reduce bleeding episodes in adults with Type 1 and 2 von Willebrand Disease (VWD), as well as on-demand and perioperative management of bleeding in pediatric patients. Previously, VONVENDI was approved for on-demand and perioperative use in adults and routine prophylaxis in adults with severe Type 3 VWD.

“Prophylactic treatment is critical to reduce the frequency of bleeds in adults, including spontaneous and joint bleeds,” said Jonathan C. Roberts, M.D., associate medical and research director at the Bleeding and Clotting Disorders Institute and associate professor of pediatrics and medicine at the University of Illinois College of Medicine at Peoria. “Establishing prophylaxis as the standard of care for all adults with VWD, regardless of subtype, represents a significant step forward in helping to manage bleeding risk and the burdens bleeds can have on patients’ daily lives.”

VONVENDI is the only recombinant von Willebrand Factor replacement therapy with approved indications in both adults and children with VWD. Designed to replace missing or dysfunctional VWF, the therapy has a uniquely long half-life (22.6 hours in adults and 14.3 hours in children), with most non-surgical bleeds in clinical trials managed with a single infusion. Approval was supported by data from three phase III trials in adults and children (NCT02973087, NCT02932618, NCT03879135) and real-world evidence.

“Children and adults living with VWD face unique challenges that can disrupt daily activities and lead to physical and emotional burdens,” said Cheryl Schwartz, Senior Vice President, U.S. Rare Disease Business Unit Lead and U.S. Commercial Operations at Takeda. “We are proud to support more patients with VONVENDI, reinforcing Takeda’s commitment to the blood and bleeding disorders community and delivering innovative therapies.”

Takeda has also submitted applications to expand VONVENDI use in pediatric patients in Japan and to approve VEYVONDI (vonicog alfa) for on-demand use in pediatric VWD patients in the European Union.

Telix Gains FDA Nod on Resubmission Pathway for TLX101-CDx NDA

Telix announced that it has reached an agreement with the FDA on the resubmission of its New Drug Application (NDA) for TLX101-CDx (Floretyrosine F18 or 18F-FET, Pixclara), an investigational agent for imaging glioma, a rare and life-threatening brain cancer. The company plans to resubmit the NDA in Q4 2025.

“As previously advised, Telix had multiple options for delivering additional data requested by the FDA in the CRL response,” said Dr. David N. Cade, Telix Group Chief Medical Officer. “This flexibility has enabled us to work efficiently to reach a mutually agreed path forward for resubmission. We remain focused on bringing this important imaging agent to patients in the U.S., supporting improved diagnosis and management of glioma.”

Following a successful Type A meeting, the FDA provided detailed feedback on the resubmission package, which will include an additional confirmatory efficacy study analysis of existing data. Telix believes this study will satisfy the FDA’s request for confirmatory evidence and address the review matters cited in the previous Complete Response Letter (CRL).

The FDA will set a new PDUFA goal date after resubmission. The agency has acknowledged the unmet medical need and indicated that expedited review is likely, subject to submission review. In the interim, Telix will continue to provide patient access to TLX101-CDx through its FDA-approved Expanded Access Program (EAP). TLX101-CDx is not included in 2025 revenue guidance, as forecasts exclude unapproved products.

Saol Therapeutics Receives FDA CRL for SL1009 in Pyruvate Dehydrogenase Complex Deficiency

Saol Therapeutics, a clinical-stage biopharmaceutical company, announced that the FDA has issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for SL1009, Sodium Dichloroacetate Oral Solution (DCA), intended for the treatment of Pyruvate Dehydrogenase Complex Deficiency (PDCD), an orphan pediatric mitochondrial disease. SL1009 has previously received Orphan Drug, Priority Review, and Rare Pediatric Disease designations from the FDA.

“Families living with PDCD face an urgent and life-threatening unmet need,” said Dave Penake, CEO of Saol Therapeutics. “We are encouraged by FDA commentary recognizing the importance of regulatory flexibility for rare diseases. Traditional clinical trials often take many years—time that children with PDCD simply do not have. We remain committed to working with the Agency to identify a path forward that brings this therapy to patients as swiftly as possible.”

PDCD affects fewer than 1,000 individuals in the U.S., with severe early-onset forms leading to high mortality. Currently, there are no FDA-approved treatments. While the CRL identified areas Saol will need to address, it did not raise any issues regarding SL1009’s manufacturing. Addressing the observations is expected to require several years and significant resources.

Saol remains encouraged by four years of clinical data from two phase III studies, including a double-blind placebo-controlled trial with ongoing open-label extension, as well as survival analysis versus a natural history cohort. Access to SL1009 will continue through both the ongoing open-label study (NCT02616484) and the Expanded Access Program (NCT06931262), which includes emergency support for neonates with life-threatening lactic acidosis due to inborn errors of metabolism.

FDA Grants Breakthrough Therapy Designation to Boehringer’s HERNEXEOS for HER2-Mutant Advanced NSCLC

Boehringer Ingelheim announced that HERNEXEOS (zongertinib tablets) has received Breakthrough Therapy Designation from the FDA for first-line treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) tyrosine kinase domain activating mutations.

“Exploring accelerated review pathways is part of Boehringer’s strategy to have a generational impact on people facing cancer,” said Vicky Brown, Senior Vice President and Head of Immunology, Oncology, and Eye Health. “We are incredibly pleased that HERNEXEOS has received Breakthrough Therapy Designation for first-line use in patients living with HER2-mutant NSCLC. This pathway highlights the potential of HERNEXEOS to address serious unmet medical needs.”

Breakthrough Therapy Designation is intended to expedite the development and review of medicines for serious conditions where preliminary clinical evidence suggests substantial improvement over existing therapies. HERNEXEOS was recently approved by the FDA for previously treated adult patients with HER2-mutant NSCLC under accelerated approval, based on objective response rate and duration of response, with continued approval contingent upon verification of clinical benefit in confirmatory trials.

The designation is supported by results from the Beamion-LUNG 1 clinical trial evaluating HERNEXEOS as a first-line treatment in HER2 (ERBB2)-mutant advanced NSCLC. Boehringer Ingelheim will present new and updated data at the upcoming World Conference on Lung Cancer (WCLC) in September and the European Society for Medical Oncology (ESMO) in October.