Ganaxolone

The FDA has signed off on Immedica Pharma's ultrarare disease treatment Loargys four years after it refused to review a marketing application for the recombinant human enzyme from another drugmaker. Four years after the FDA issued its most heavy-handed form of a rejection to the prior company behind pegzilarginase, the U.S. regulator has now given the treatment a thumbs-up.Scoring the accelerated nod is Immedica Pharma for Loargys as a therapy for hyperargininemia in the ultrarare genetic disorder Arginase 1 deficiency (ARG1-D). The approval covers patients age 2 and older, with the therapy to be used in conjunction with a protein-restricted diet. Loargys, which is also known as pegzilarginase, is a recombinant human enzyme designed to lower levels of arginine in patients who are unable to break down the amino acid. It is the first treatment to address the elevated levels of plasma arginine associated with the disorder. The approval also comes after the FDA issued a complete response letter (CRL) to the company in August of last year citing major deficiencies across efficacy, dosing/administration, safety and labeling.ARG1-D affects an estimated 250 people in the U.S., with the current standard of care relying on symptom management including diet restrictions, arginine-free amino acid supplementation and nitrogen scavenging agents. Patients are often diagnosed in late infancy or early childhood, and the symptoms include spasticity, seizures and intellectual disability. “The accelerated approval of Loargys offers a fundamentally new approach that addresses the enzyme deficiency itself,” Stephen Cederbaum, M.D., a professor of human and medical genetics at the University of California, Los Angeles, said in a release. “Since persistently elevated arginine and its metabolites have been reported to be the proximal or direct driver of disease progression, this is a major advancement in metabolic medicine.” The company expects to make Loargys—which is infused or injected weekly—available in the U.S. in April. The green light comes after the FDA dismissed a marketing application for pegzilarginase in 2022 from Texas-based Aeglea BioTherapeutics, handing down a refusal-to-file (RTF) letter. An RTF is issued when the agency determines an application cannot be reviewed due to deficiencies that cannot be quickly resolved.A year after the rejection, Aeglea sold pegzilarginase to its European partner Immedica for $15 million upfront and $100 million in potential milestones.While a clinical trial of pegzilarginase linked it to an 80% reduction in mean plasma arginine compared to placebo, there were questions about whether it improved the health of patients. While mobility scores for patients improved in the treatment arm and worsened in the placebo group, the difference fell short of statistical significance. To ultimately gain FDA approval of Loargys, Immedica relied on results from the same trial, while also submitting additional "long-term data," it said in an email. "The application was resubmitted with additional data, analyses and clarifications addressing each of the FDA’s objections in the refusal to file and advice in regulatory interactions, which ultimately supported the agency’s decision to accept the filing for review," the company added.Additionally, in the August CRL, the FDA suggested the possibility of an accelerated-approval pathway "only with a postmarketing confirmatory trial designed to validate surrogate endpoints and demonstrate clinical benefit."In December 2023, Sweden-based Immedica earned marketing approval of Loargys from the European Medicines Agency. The private company, which was established in 2018 by Nordic investment firm Impilo, does not report sales of its products.At the end of 2024, Immedica acquired troubled Marinus Pharmaceuticals and its seizure medicine Ztalmy, which was approved in 2022.EDITOR'S NOTE: Immedica's responses to questions about how it ultimately gained FDA approval of Loargys were added to the story after it was initially published.

First-ever approved treatment for CDD in China now set for commercial launch through dedicated rare disease platform SHANGHAI--(BUSINESS WIRE)-- Tenacia Biopharmaceuticals (Shanghai) Co., Ltd. (" Tenacia ") and GOLDEN AGE HEALTH (SHANGHAI) CO., LTD., a subsidiary of Golden Age Health Pte. Ltd. (collectively, " GAH ") today announced a Commercialization Agreement granting GAH exclusive rights to commercialize ZTALMY ® (泽元安), the first and as of now only approved treatment for CDKL5 deficiency disorder (CDD) in Mainland China. ZTALMY ® (ganaxolone oral suspension) was developed by Marinus and Tenacia obtains exclusive rights to develop and commercialize it in Greater China. In July 2024, the drug was approved by the National Medical Products Administration for treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. Tenacia has partnered with GAH to leverage its comprehensive patient-focused commercialization platform—encompassing disease awareness, diagnosis support, market access, and patient affordability programs—to ensure this therapy reaches the families affected by this devastating rare epilepsy. "Bringing the first approved therapy for CDKL5 deficiency disorder to patients in China represents exactly the type of mission-critical work Golden Age Health was built for," said Chao (Brook) Wu, Chief Executive Officer of Golden Age Health (Shanghai) . "We are honored to partner with Tenacia to ensure that children and families affected by CDD can access this breakthrough treatment as quickly as possible." Dr. Xiaoxiang Chen, Founder and CEO of Tenacia Biopharmaceuticals (Shanghai) , said: "GAH's proven expertise in rare disease commercialization and their commitment to patient access make them the ideal partner to bring ZTALMY ® to the patients who need it most in China. Together, we will work to maximize the impact of this important therapy." About CDKL5 Deficiency Disorder CDKL5 deficiency disorder (CDD) is a rare developmental epileptic encephalopathy caused by mutations in the CDKL5 gene. Patients typically experience early-onset, treatment-resistant seizures, profound developmental delays, and motor impairments. CDD is included in China's National Rare Disease Catalog , reflecting the urgent need for effective treatments for affected patients and their families. About ZTALMY ® ( ganaxolone oral suspension) ZTALMY ® (ganaxolone oral suspension) is a neuroactive steroid GABA A receptor positive allosteric modulator. The oral suspension formulation (110ml: 5.5g) is approved by China's NMPA for the treatment of seizures associated with CDKL5 deficiency disorder in patients 2 years of age and older. It represents the first therapy specially approved for this indication in China. About Golden Age Health Golden Age Health is a specialty pharmaceutical company dedicated to redefining patient access to medicine across China, Asia-Pacific and MEA. Headquartered in Singapore, GAH pairs data-driven market-access expertise with deep medical-affairs and RWE capabilities to bring high-impact therapies to underserved populations. About Tenacia Biopharmaceuticals Founded in 2022 by Bain Capital, Tenacia is a commercial-stage biopharmaceutical company dedicated to developing innovative therapeutics for patients with underserved neurological disorders. Tenacia's seasoned management team brings decades of drug development experience from both MNCs and biotechs. Their deep understanding of complex disease biology and insights into unmet patient needs enable Tenacia to create a highly efficient neuroscience R&D platform with a proven track record of advancing novel therapies. Empowered by its founder and strategic investor, Bain Capital – through significant initial investment, long-term commitment and extensive life science network – Tenacia is poised to become a leading neuroscience company in China and deliver substantial value to shareholders while upholding an unwavering commitment to the patients. Forward-Looking Statements This release contains forward-looking statements, including expectations regarding clinical development, regulatory matters and commercial launches. These statements involve risks and uncertainties that may cause actual outcomes to differ materially. Contacts Media & Investor Contacts Golden Age Health Media/IR: info@gahbioventures.com Tenacia Biopharmaceuticals 官网：