Jazz Pharmaceuticals Plc

FRIDAY, Feb. 6, 2026 -- For early-morning shift workers with shift work disorder (SWD), treatment with solriamfetol significantly improves sleepiness, according to a study published in the February issue of NEJM Evidence . Kirsi-Marja Zitting, Ph.D., from Brigham and Women's Hospital in Boston, and colleagues conducted a randomized trial to evaluate the effectiveness of solriamfetol, a selective dopamine and norepinephrine reuptake inhibitor, for treating excessive sleepiness in 78 early-morning shift workers (shift starting between 3 and 7 a.m.), with SWD-associated excessive sleepiness. The researchers found that compared with patients treated with placebo, those treated with solriamfetol were significantly less sleepy after four weeks (9.4-minute longer sleep latency). Solriamfetol treatment was associated with altered sleepiness (Karolinska Sleepiness Scale difference, −1.2) and changes in clinician and patient ratings of the change in clinical condition (odds ratios, 3.7 and 4.2, respectively). Overall, 55 and 63 percent of patients who received any treatment with solriamfetol and placebo, respectively, reported any adverse event; headache and nausea were the most common adverse events. "The improvement we saw is clinically meaningful. These workers were able to stay awake and alert throughout a full eight-hour shift, which has real implications for performance, safety, and quality of life," coauthor Charles A. Czeisler, Ph.D., M.D., also from Brigham and Women's Hospital, said in a statement. "Shift workers are essential to how our society functions, yet they often pay a hidden biological cost. This study shows we can do better for them." Several authors disclosed ties to biopharmaceutical companies, including Jazz Pharmaceuticals and Axsome Therapeutics; Axsome manufactures solriamfetol and partially funded the trial. Abstract/Full Text (subscription or payment may be required) Editorial (subscription or payment may be required)

The programme’s return is being authorised under the Mikaela Naylon Give Kids a Chance Act, a bill that addresses shortcomings in paediatric rare disease and cancer drug development. Mary Long via Shutterstock.com. After a lengthy period in regulatory limbo, the US Food and Drug Administration’s (FDA) rare paediatric disease priority review voucher (PRV) programme has returned to the pharma landscape, marking a win for biopharma companies developing therapies for rare diseases in children. On 3 February, President Donald Trump signed a spending bill that will fund several federal agencies for the remainder of 2026. Included in the bill was the rare paediatric disease PRV programme, which has been inactive since it expired in December 2024. The programme’s return is being authorised under the Mikaela Naylon Give Kids a Chance Act, a bill that covers a range of regulatory frameworks to increase the speed at which children battling rare diseases and certain cancers can access therapies. Jeremiah Kelly, partner at law firm Venable, told Pharmaceutical Technology: “ The reauthorisation of the pediatric priority review voucher program will incentivise drug and biologics development for diseases that, otherwise due to low prevalence, lack the return-on-investment in the market to compel companies to undertake the costly risks associated with that development.  Thankfully, Congress has reauthorised this program, which should strengthen the pipeline of products on the way to help some of our most vulnerable children – this program shifts the balance more in favor of innovation in the pediatric drug development space.” First put forward by Republican Congressman Michael McCaul in February 2025, the Mikaela Naylon Give Kids a Chance Act combines two initiatives – the original Give Kids a Chance Act and the Creating Hope Reauthorization Act. The aim of the legislation is to spur drug development for paediatric rare diseases, improve outcomes for patients and close gaps in paediatric drug research. The Act has received bipartisan support during its journey from Congress to Trump’s signature. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Programme’s return welcomed The rare paediatric disease PRV programme expired over a year ago due to political and congressional delays . Patient advocacy groups and pharma industry bodies continually called for its reauthorisation, given the framework’s importance in bringing innovative therapies to market. Many of these groups have now welcomed the programme’s return to the US pharma scene. The Rare Disease Company Coalition said in a statement: “[We] are pleased to celebrate the passage of the Mikaela Naylon Give Kids a Chance Act, which reauthorises the lifesaving paediatric rare disease PRV programme.” “This milestone brings renewed hope to families awaiting lifesaving treatments for children with rare diseases and reaffirms a bipartisan commitment to advancing innovation for paediatric patients who urgently need therapeutic breakthroughs. The PRV program has already helped enable therapies across 47 rare paediatric indications,” added the coalition, of which BioMarin and Sarepta Therapeutics are members. It is currently unclear when the FDA will resume reviewing requests for designation and if there will be any amendments to the scheme’s regulatory process. According to the National Organization for Rare Disorders (NORD), 63 PRVs have been awarded across 47 rare paediatric diseases since the programme’s inception. Prior to its creation, only four of these 47 rare paediatric diseases, many of which lead to death before the patient reaches adulthood, had a treatment approved by the FDA. “The reauthorisation of the PRV programme and the accompanying bills will speed up innovation and access, ensuring that more patients have a chance at a life not limited by rare disease,” the NORD said in a statement. Rare disease incentive back on the agenda Companies that receive FDA approval for a drug or biological product for a rare paediatric disease can qualify for a voucher that can be redeemed to receive priority review for a different product.  The voucher slashes the FDA review time for a new drug application from the standard 10 months to 6 months, allowing pharma companies to recoup R&D expenses in a more lucrative market. Companies are also able to sell vouchers for cash. Since the PRV framework was installed in 2014, a secondary market of vouchers has established itself. In January 2026, Jazz Pharmaceuticals announced it had sold a voucher for $200m . Other sales in 2025 hovered around the $150m price point . The incentive means more rare diseases are pursued as part of pharma pipelines. SynaptixBio, a biotech developing rare disease drugs, currently holds two rare paediatric disease designations. The company’s CEO, Dan Williams, says the bill’s passage is significant for smaller biotechs. Williams stated: “The PRV programme means smaller biotechs can see a way to financial security, having typically had to find investment during the long years of drug design and development. “The very high cost of preparing for, and conducting, clinical trials adds to the financial challenge, and all the risk remains until a candidate drug is approved for the market… big pharma doesn’t face the high investment risk of developing a rare disease drug, and the smaller biotech has a natural target for selling its PRV.” In June 2025, the FDA rolled out a pilot scheme for a new type of priority voucher. This new framework, called the Commissioner’s National Priority Voucher (CNPV) programme, is available to companies developing a drug with US national interests as a primary focus. While rare diseases can fulfil the criteria, they are not the exclusive target of the initiative. Vouchers awarded under the scheme will shorten FDA review time for a new drug application from 10-12 months to up to two months.

Exxel is betting that EX937 will sidestep the side effects that plagued another high-profile FAH inhibitor.\n Exxel Pharma is eyeing a listing on the NYSE American to fund human trials for a drug class that has previously struggled to deliver in the clinic.The Colorado-incorporated company set out plans to sell 3 million shares at $5 apiece. Exxel estimates the IPO could bring in $13.2 million, according to a Securities and Exchange Commission filing submitted Jan. 30.At the top of the list of spending priorities is an estimated $80,000 that Exxel has earmarked to manufacture and test sufficient quantities of its lead candidate, EX937, with the goal of taking the drug into a phase 1 trial in Australia this year. A further $3.8 million will be set aside for the study itself.EX937 is a fatty acid amide hydrolase (FAAH) inhibitor in development for refractory chronic cough and other hypersensitivity disorders that share the pathophysiology of hypersensitized sensory neurons. The aim is to make EX937 into a “pipeline-in-a-drug” via phase 2 trials for indications like hyperactive bladder, painful peripheral neuropathies, and migraine headache, according to Exxel. FAAH helps regulate the body\'s cannabinoid receptors, making it a seemingly promising target for the treatment of pain and anxiety. But biopharmas have struggled to demonstrate this promise in the clinic.Back in 2016, a FAAH inhibitor anxiety treatment from Portuguese biotech Bial led to six hospitalizations and one death in a phase 1 study. That result persuaded Johnson & Johnson to abandon its own FAAH inhibitor for depression, while Jazz Pharmaceuticals dropped a FAAH inhibitor after a phase 2 fail in post-traumatic stress disorder in 2023.EX937—which Exxel licensed from the University of California, Irvine—is “actively excluded from the central nervous system,” according to the company. The biotech is betting that this will “greatly reduce the potential for centrally mediated side effects, and differentiates its mechanism of action from FAAH inhibitors of the past.”As well as EX937, the company has also licensed other FAAH inhibitors from the university. Exxel is hoping these compounds “have applications in multiple CNS disorders, including social anxiety disorder and autism spectrum disorder, which are at present untreated.”“Exxel is currently evaluating options for effectively commercializing [these compounds] without distracting resources from the lead asset EX937,” the company explained in the SEC filing. The biotech has been led by Soren Mogelsvang, Ph.D.—the company’s only full-time employee—since 2017. Mogelsvang previously served as CEO of veterinary health company Peak Pharmaceuticals and, since 2021, has juggled his Exxel responsibilities with serving as director for neuropsychiatric and autoimmune biotech MycoBiotix.Heading up Exxel’s R&D is Chief Scientific Officer Daniele Piomelli, Ph.D., M.D., who concurrently serves as CEO of Nectaris, a company focused on developing non-pharmaceutical treatments for metabolic syndrome and chronic pain, according to the filing. Piomelli, along with the company’s Chief Medical Officer, Richard Paul, M.D., and Chief Financial Officer, Robert Dickey IV, were Exxel’s only other employees, and all three held part-time positions as of last September.