The FDA approval was supported by data from the most extensive Phase III pivotal trial program conducted in CF, involving over 1,000 patients across 20 countries. The studies demonstrated that ALYFTREK met its primary and key secondary endpoints, showing non-inferiority to TRIKAFTA in lung function (ppFEV1) and statistically significant improvements in sweat chloride (SwCl) levels. ALYFTREK was generally well-tolerated, with safety confirmed across all age groups, including children aged 6-11 years.
Dr. Claire L. Keating, Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University, emphasized the significance of the approval, stating, “In Phase III clinical trials, once-daily ALYFTREK demonstrated non-inferiority to TRIKAFTA in ppFEV1 response and statistically significant improvement in SwCl, a welcomed advancement for the treatment of CF. ALYFTREK has the potential to improve the care of patients with CF.”
As the first once-daily CFTR modulator, ALYFTREK addresses a key unmet need in CF treatment. Approximately 75% of surveyed physicians have highlighted the importance of more convenient dosing for people with CF, who must take modulators with fat-containing food. Additionally, ALYFTREK offers a transformative option for 150 people in the U.S. with one of 31 mutations newly eligible for a CFTR modulator. The therapy has also been submitted for regulatory approval in the European Union, the United Kingdom, Canada, Switzerland, Australia, and New Zealand, signaling its potential for global impact.
FDA Approves ZEPBOUND as the First Treatment for Obstructive Sleep Apnea in Obese Adults
Eli Lilly and Company has received FDA approval for ZEPBOUND (tirzepatide), the first and only prescription medication to treat adults with moderate-to-severe obstructive sleep apnea (OSA) and obesity. ZEPBOUND, designed to improve sleep disorder symptoms, is intended for use alongside a reduced-calorie diet and increased physical activity.
“Too often, OSA is brushed off as ‘just snoring’ — but it’s far more than that,” said Julie Flygare, J.D., president and CEO of Project Sleep. “It’s important to understand OSA symptoms and know that treatments are available, including new options like ZEPBOUND. We hope this will spark more meaningful conversations between patients and health care providers and ultimately lead to better health outcomes.”
OSA, a serious condition characterized by partial or complete upper airway collapses during sleep, can lead to pauses in breathing (apnea) and reduced oxygen levels. The disorder often goes undiagnosed, despite symptoms such as snoring, daytime fatigue, and disrupted sleep.
“Today, many cases of OSA go undiagnosed and untreated, leaving millions at risk for serious health consequences,” noted Patrik Jonsson, executive vice president, and president of Lilly Cardiometabolic Health and Lilly USA. “ZEPBOUND is the first medication that significantly improves moderate-to-severe OSA and aids in long-term weight loss in adults with obesity. Nearly half of clinical trial patients saw such improvements that they no longer had symptoms associated with OSA.”
The FDA’s decision follows the results of the SURMOUNT-OSA Phase IIIK clinical trials, where ZEPBOUND demonstrated superior efficacy compared to placebo. Patients on ZEPBOUND without positive airway pressure (PAP) therapy experienced a reduction of 25 breathing disruptions per hour compared to five with placebo. With PAP therapy, ZEPBOUND reduced 29 disruptions per hour compared to six with placebo. Moreover, ZEPBOUND led to substantial weight loss, with participants losing an average of 18-20% of their body weight, depending on their PAP usage.
ZEPBOUND contains tirzepatide and comes with safety considerations, including risks related to thyroid tumors. Patients and providers should discuss potential side effects and pre-existing conditions, such as a history of medullary thyroid carcinoma or Multiple Endocrine Neoplasia syndrome type 2. With its dual impact on OSA symptoms and obesity, ZEPBOUND offers a transformative treatment option for a condition affecting millions.
Tonix Pharmaceuticals’ TNX-102 SL NDA Accepted by FDA for Fibromyalgia Treatment
Tonix Pharmaceuticals Holding Corp. announced that the FDA has accepted the New Drug Application (NDA) for TNX-102 SL (cyclobenzaprine HCl sublingual tablets), a non-opioid, centrally-acting analgesic for managing fibromyalgia. With this acceptance, the FDA is expected to issue a Day 74 Letter, which will include a Prescription Drug User Fee Act (PDUFA) target action date and indicate whether Priority Review has been granted. Notably, TNX-102 SL was granted Fast Track designation by the FDA in July 2024, a status intended to expedite the review of innovative treatments addressing unmet medical needs.
“The FDA’s acceptance of our NDA represents another step forward as we pursue our goal of delivering the first member of a new class of medicines for the management of fibromyalgia, a condition affecting over 10 million adults in the U.S.,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “The fibromyalgia community, comprised of patients and their families and support groups, has been waiting for a new drug for over 15 years. Analysis of insurance claims in the U.S. has shown that 18 months after diagnosis, fibromyalgia patients are more likely to be prescribed addictive opioids than all three FDA-approved drugs combined.”
Dr. Lederman added, “We look forward to working closely with the FDA throughout the NDA review process to bring TNX-102 SL to the market and address the significant unmet needs of the fibromyalgia community as quickly as possible. This milestone also marks an important step as we advance our commercial preparations in anticipation of potential approval in 2025. With a strong commercial leadership team in place, we are prepared to support this launch alongside our marketed migraine treatments, Zembrace® SymTouch® and Tosymra®.”
The NDA submission is supported by data from two Phase III clinical trials, RELIEF and RESILIENT, which demonstrated that TNX-102 SL significantly reduced daily pain compared to placebo in fibromyalgia patients. Both trials met their primary endpoints with robust statistical significance and showed that the treatment was well tolerated, with no new safety concerns. The most common adverse event was transient tongue or mouth numbness, which was self-limited and rarely led to discontinuation. Tonix believes this comprehensive data package provides the necessary evidence to support FDA approval.
Quoin Pharmaceuticals Gets FDA Clearance for QRX003 Netherton Syndrome Study
Quoin Pharmaceuticals Ltd. announced that the FDA has cleared a new clinical study for QRX003 in Netherton Syndrome. QRX003 is a topical lotion containing a broad-spectrum serine protease inhibitor designed to target kallikreins in the skin, the enzymes responsible for the excessive skin shedding associated with Netherton Syndrome. This new study represents the most extensive use of QRX003 in a clinical setting to date. It will involve applying the product to over 80% of participants’ body surface area (BSA) twice daily for 12 weeks.
Dr. Amy Paller of Northwestern University, who will lead the study, emphasized the innovative approach of QRX003. “The best kind of treatment, short of curative gene therapy, focuses on reversing the mechanism by which skin disease occurs,” said Dr. Paller. “Targeting kallikreins, which are thought to lead to the clinical manifestations of Netherton Syndrome, could be an ideal approach.”
Quoin’s CEO, Dr. Michael Myers, highlighted the significance of this development in advancing treatment options for NS. “Following our recent announcement of positive interim clinical data from two of our ongoing Netherton Syndrome clinical studies, we are very excited to announce FDA clearance for this groundbreaking additional study for QRX003,” he stated. “We are extremely pleased to be working with Dr. Paller and look forward to generating real-world data, which will be a key component of our regulatory filings. This reflects our unwavering commitment to developing the first-ever approved treatment for this devastating disease.”
The study will supplement data from Quoin’s ongoing open-label and double-blinded clinical trials, where QRX003 is applied to approximately 20% of participants’ BSA. This new research aims to closely replicate real-world application scenarios for NS patients, significantly enhancing the dataset to support potential regulatory approval of QRX003.
FDA Greenlights First Mesenchymal Stromal Cell Therapy for Steroid-Refractory Acute GvHD
The FDA has approved Ryoncil (remestemcel-L-rknd), the first mesenchymal stromal cell (MSC) therapy for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged two months and older. Ryoncil is an allogeneic (donor-derived) bone marrow-based therapy, containing MSCs isolated from healthy adult donors’ bone marrow. These cells possess the ability to differentiate into various cell types and play diverse roles in the body.
“Today’s decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children,” stated Peter Marks, M.D., Ph.D., Director of the FDA’s Center for Biologics Evaluation and Research (CBER). “This first mesenchymal stromal cell therapy approval demonstrates the FDA’s commitment to supporting the development of safe and effective products that could improve the quality of life for patients with unresponsive symptoms to other therapies.”
SR-aGVHD is a severe, life-threatening complication that can follow allogeneic hematopoietic stem cell transplantation (allo-HSCT). This procedure is often performed to treat specific blood cancers, blood disorders, or immune system conditions. SR-aGVHD involves significant health consequences, including damage to multiple organs and a high risk of death. “The FDA remains dedicated to helping address the urgent unmet needs of individuals with debilitating and deadly diseases, and today’s approval is an important step in that effort,” said Nicole Verdun, M.D., Director of the Office of Therapeutic Products in CBER.
Ryoncil’s approval is based on a multicenter, single-arm study involving 54 pediatric participants with SR-aGVHD. Participants received eight intravenous infusions of Ryoncil over four weeks, with additional doses for partial or mixed responders. Results showed a complete response in 30% of patients and a partial response in 41% by day 28, demonstrating its potential effectiveness. Adverse reactions included infections, fever, abdominal pain, and hypertension, among others. The FDA advises monitoring during infusion to manage potential reactions and hypersensitivity risks.
