Xenon Pharmaceuticals, Inc.

iStock, Mikhail Makarov The House Committee on Energy and Commerce has cleared proposed legislation that could bring back the FDA’s rare pediatric priority review voucher program, which allows for expedited drug reviews. The FDA’s rare pediatric priority review vouchers are one step closer to becoming available to drugmakers again, after the House Committee on Energy and Commerce advanced a bill that would restart the program. Lawmakers on Wednesday ushered the proposed legislation out of committee on Wednesday, according to reporting from Endpoints News . If it passes full House and Senate votes, this bill , dubbed the Give Kids a Chance Act of 2025, would keep the program alive through Sept. 30, 2029. The proposal will now move forward to be discussed and voted on by the entire House. First enacted in 2012, the FDA’s rare pediatric priority review voucher (PRV) program is designed to incentivize drugmakers to work on rare diseases afflicting children. Companies that successfully secure an approval for a therapy treating a rare disease are then awarded a voucher that, if claimed, will qualify another of their products for priority review. Sponsors can also sell their vouchers to another sponsor for often much-needed revenue. In September 2024, however, Congress failed to authorize the renewal of the program, forcing the FDA to “ sunset ” the system after Dec. 20, 2024. Speaking to BioSpace in March this year, Matthew Winton, chief operating officer of Inozyme Pharma, said the program had bipartisan support, but that it “got caught” in between timelines and changing priorities. The consequences of this failure to keep the program running were immediate and far-reaching. According to a BioSpace analysis in March, pediatric rare disease vouchers accounted for the vast majority of these fast-track tickets that the FDA hands out in a year. In 2024, for instance, the agency issued 11 vouchers—the most of any year since the program was launched—10 of which were connected to pediatric rare disease development. After the program went offline, many companies specializing in rare disease were forced to warn investors that may never receive their vouchers. Among these are Xenon Pharmaceuticals , OS Therapies , Amylyx Pharmaceuticals and Solid Biosciences . These rare disease PRVs, according to Winton, “feed the cycle of innovation and bringing drugs to patients in the U.S. who have no other options.”

SK Biopharmaceutical hopes to cross the $1 billion sales marker for Xcopri by 2029. SK Biopharmaceutical’s Xcopri (cenobamate) met the bar in a phase 3 trial that could support the company’s ambitions of building a blockbuster with an expansion into another seizures subtype.The South Korean pharma won FDA approval for Xcopri in 2019 as a treatment for partial-onset seizures, also known as focal seizures, in adults. Since then, with rapid global growth, SK has outlined blockbuster ambitions for the drug by 2029. Now, with a positive phase 3 trial, another indication for primary generalized tonic-clonic (PGTC) seizures could be on the table.  In its phase 3 study, SK evaluated Xcopri in 169 adult and adolescent patients with PGTC seizures, finding a “statistically significant reduction” in the seizures, the company said in a filing on the Korea Exchange. Specifically, seizure frequency from baseline levels was reduced by 71.9% in the Xcopri-treated group and 39.6% in the placebo arm over 28-day intervals.PGTC seizures are the most common type of generalized seizures—which are less prevalent than focal seizures—and considered the most severe due to the high risk of seizure-related injury and sudden unexpected death in epilepsy, SK pointed out. With limited treatment options, the company hopes Xcopri can address an unmet need and contribute to quality-of-life improvements for epilepsy patients, it said.While the current treatment landscape for the seizure type is sparse and generally made up of antiseizure medications such as GSK’s off-patent Lamictal, SK isn’t the only one looking to edge in on the market. Xenon Pharmaceuticals is approaching an early 2026 phase 3 readout of its lead candidate, azetukalner, in focal seizures as an additional phase 3 trial in PGTC seizures continues.According to a note by Jefferies analysts, SK’s win in PGTC seizures “serves as a reminder” that Xenon’s azetukalner could show “broad-spectrum activity” as well, supporting peak sales expectations of $400 million from that indication alone and $1 billion on the focal seizures side. However, SK still has some breathing room from the competition as the analysts note that SK’s study took seven years to produce top-line data after its 2018 start, suggesting that Xenon’s phase 3 could take “several years” as well.SK will file for a label expansion with the FDA and present additional analysis from the study at the American Epilepsy Society Annual Meeting in December. Last quarter, Xcopri helped the company reach record sales with U.S. revenues of $111 million. 

Dive Brief:The Food and Drug Administration on Wednesday issued draft recommendations for drugmakers interested in developing new non-opioid options to treat chronic pain.The document lays out the agencys expectations for clinical trials and includes recommendations for seeking broad indications for pain that cover multiple conditions instead of just one. It also details how drugmakers should test their experimental medicines to see if they can help patients reduce or replace opioids or avoid starting on the drugs altogether.Agency officials are open to offering expedited clearance pathways to spur the development of non-opioid medicines, according to the guidance. Still, the FDA warned that it may be difficult to use the accelerated approval program because pain is so personal that its difficult to envision how surrogate or intermediate endpoints could be used to predict analgesic effect.Dive Insight:While their addictive properties are now well known, opioids remain commonly prescribed because they are effective at neutralizing pain and often available in cheap, generic form. About one out of every five adults suffering from chronic pain takes opioids, according to the FDA.Efforts to develop alternatives have been hampered by the significant challenges in pain research. Scientists dont fully understand the mechanisms behind pain and analgesia, and two people with the same injury can have completely different experiences. In addition, compounds that look promising in laboratories often fail to translate to safe and effective medicines for people.The FDA is hoping to kickstart more research by making regulatory pathways more predictable for innovators and drug developers, with the guidance, FDA Commissioner Marty Makary said in a statement accompanying the guidance. Physicians need more alternatives.There are signs of promise in the industry. Vertex Pharmaceuticals in January won approval for Journavx, a first-of-its-kind, non-opioid medicine to treat acute episodes of pain, often experienced after an accident or surgery. Several other biotechs are looking to develop similar treatments.The FDA guidance should help spur more interest in the industry, Jefferies analyst Andrew Tsai said in a note to clients. We sense a high degree of FDA interest in helping sponsors develop non-opioid approaches, Tsai wrote. He said he expects companies, including Xenon Pharmaceuticals and Rapport Therapeutics, to consider exploring the FDAs expedited pathways for review.The agency issued the guidance to meet a requirement in the SUPPORT Act, a law designed to help patients and communities coping with the opioid crisis. '