Regulatory – Life Sciences Voice

FDA Sets Tougher Approval Criteria for Covid Booster Shots in Healthy Individuals

Editorial Team — Thu, 22 May 2025 19:57:51 +0000

The Food and Drug Administration (FDA) has introduced more rigorous regulatory requirements for approving future COVID-19 booster shots, particularly for healthy individuals. This marks a significant shift from the previous approach, which typically approved annual boosters for all Americans based mainly on lab tests showing a strong immune response. Under the new guidance, pharmaceutical companies must now conduct clinical trials to prove that updated boosters are both safe and effective in healthy adults and children. This change could reduce the number of people eligible for routine boosters and increase costs for vaccine manufacturers.
Dr. Vinay Prasad, a vocal critic of pharmaceutical industry practices and now head of the FDA’s vaccine division, expressed skepticism about the blanket recommendation for repeated boosters in healthy individuals. He emphasized the lack of clear evidence supporting multiple annual doses for low-risk Americans and advocated for a more targeted approach.

In a paper published in the New England Journal of Medicine, FDA Commissioner Marty Makary and Dr. Prasad outlined a new risk-based strategy. For high-risk groups—including people over 65 and those with certain medical conditions such as obesity or depression—the FDA will continue to accept immunogenicity data as sufficient for approval. These individuals, estimated to number between 100 to 200 million, would still have relatively easy access to new vaccines.
However, for healthy individuals aged six months to 64 years without underlying health issues, the FDA will now require evidence from randomized, placebo-controlled trials. These trials must demonstrate that boosters reduce symptomatic COVID-19 cases by at least 30%, and participants must be monitored for at least six months to assess lasting protection. The new approach reflects the agency’s view that annual updates for all Americans are no longer scientifically justified, especially as the virus is now mutating more slowly than influenza.
The FDA stated that while it will continue to support rapid access for vulnerable populations, it expects drugmakers to conduct more thorough post-market studies in healthy groups. This approach seeks to strike a balance between ensuring safety and avoiding unnecessary vaccinations.

Financial analysts responded cautiously to the news. While acknowledging potential impacts on revenue for vaccine makers like Pfizer, BioNTech, and Moderna, they described the FDA’s stance as reasonable and science-based. Some noted that the new risk-based model aligns with international practices, where most high-income countries limit COVID booster recommendations to older or high-risk individuals.
The FDA’s paper also criticized the previous “one-size-fits-all” strategy, which it said may have eroded public trust in vaccination. Citing CDC data, it noted declining booster uptake, especially among children and even healthcare workers. The authors argued that unnecessary annual COVID shots could contribute to broader vaccine hesitancy, including for well-established vaccines like MMR (measles, mumps, rubella), which remain vital.Ultimately, the FDA’s revised guidance represents a shift toward a more evidence-driven, personalized approach to COVID-19 vaccination, with future approvals grounded in stronger data and differentiated by patient risk.

FDA Delays Biohaven’s Approval Decision for Rare Disease Drug Troriluzole

Editorial Team — Tue, 20 May 2025 23:17:38 +0000

The U.S. Food and Drug Administration (FDA) has postponed its decision on Biohaven Pharmaceuticals’ lead drug candidate, troriluzole, a potential treatment for a group of rare neurological conditions that damage nerve function. The company had anticipated a regulatory verdict by the end of September, following the completion of late-stage clinical trials. However, Biohaven announced that the FDA now requires additional time and input from an independent advisory committee, a move that adds uncertainty to the review process.

This development contradicts Biohaven’s previous communication. In its recent earnings report, the company stated that FDA officials had not mentioned the possibility of convening an advisory panel—a step usually taken when the agency has remaining questions about the safety, efficacy, or overall benefit-risk profile of a drug under review.

Troriluzole is Biohaven’s most advanced clinical candidate and is designed to metabolize into a molecule already approved in the U.S. and Europe to treat amyotrophic lateral sclerosis (ALS). Biohaven is positioning the drug as a therapy for a wider range of conditions, from the rare spinocerebellar ataxia (SCA) to more common illnesses like obsessive-compulsive disorder (OCD).

Key Developments and Market Impact

Regulatory setback in the U.S.: The FDA’s request for an advisory committee adds months to the review process and suggests increased regulatory scrutiny.
Unexpected reversal: Just days before the announcement, Biohaven reported no signs from the FDA of needing such a meeting.
European withdrawal: In March, Biohaven withdrew its marketing application for troriluzole in Europe after the EMA indicated likely rejection and declined to grant a special designation with economic benefits.
Market reaction: The European decision alone led to a $400 million loss in Biohaven’s market value. Combined with other setbacks, the stock is down roughly 15% over the past six months.
Investor concerns: Analysts such as Leonid Timashev of RBC Capital Markets say the new delay raises doubts about the overall regulatory outlook for troriluzole and increases the chances of a rejection.
Staffing issues at the FDA: Some believe the delay may also be partly attributed to internal staffing shortages at the FDA following recent layoffs.

If successful, troriluzole would become the first approved treatment for SCA and could mark a turning point for Biohaven, which has not brought a new therapy to market since it sold its migraine drug portfolio to Pfizer for $12 billion in 2022. The company has indicated it plans to reapply in Europe and remains committed to advancing its rare disease pipeline.

Abeona Nets $155M in Voucher Sale After FDA Approval

Editorial Team — Thu, 15 May 2025 05:22:28 +0000

Abeona Therapeutics has secured a $155 million deal from the sale of a Priority Review Voucher (PRV), just two weeks after receiving U.S. Food and Drug Administration (FDA) approval for its first commercial product. The company did not disclose the name of the buyer.
The PRV was awarded following the FDA’s approval of Zevaskyn, a gene therapy developed to treat recessive dystrophic epidermolysis bullosa (RDEB), a rare and serious skin disorder. Zevaskyn is an autologous, cell-based therapy that uses a patient’s own genetically modified skin cells to produce functional Type VII collagen. It is applied as a sheet to wound areas and is designed to aid in healing chronic wounds and alleviating pain associated with RDEB.

Priority Review Vouchers are issued by the FDA as an incentive to encourage the development of treatments for rare or neglected diseases. These vouchers allow the holder to expedite the review process of another drug application. For companies developing competitive or time-sensitive therapies, the shortened review period can be of considerable value. Recent PRV sales in the industry have ranged between $150 million and $158 million, placing Abeona’s transaction within the typical market range.
In a press release issued Monday, Abeona stated that the proceeds from the sale position the company with enough operating capital for over two years without requiring additional funding or factoring in revenue from Zevaskyn. Chief Financial Officer Joe Vazzano noted that the company expects to reach profitability in early 2026.
Zevaskyn is scheduled for commercial launch in the third quarter of 2025. Abeona has indicated a phased rollout strategy, with an initial projection of 10 to 15 patients treated in the first year. This estimate reflects a deliberate approach to adoption, which the company anticipates will accelerate over time.
Abeona CEO Vishwas Seshadri discussed the rationale behind the timing of the PRV sale. He cited continued demand for such vouchers, with the last four PRV transactions in the industry all closing at $150 million or more. Seshadri emphasized the company’s intent to complete the transaction quickly in light of regulatory uncertainties surrounding the PRV program.

The sale of the PRV also comes at a time when Abeona faces broader market challenges. The company has experienced pressure from a depressed stock price, limiting its ability to raise capital through equity. The $155 million infusion from the voucher sale provides an alternative financial strategy to support operations and the commercial development of its approved product.
Zevaskyn will enter a therapeutic space that includes other treatment options for epidermolysis bullosa, such as a weekly topical gel from Krystal Biotech. Unlike its competitor, Zevaskyn is a one-time treatment, which differentiates its method of delivery and application.
Following the announcement of the PRV sale, Abeona’s stock rose approximately 10%, reaching about $6 per share in early trading. The transaction underscores a shift in Abeona’s operational strategy as it transitions from development-stage biotech to a commercial-stage company.

FDA and CDC Recommend Halting Chikungunya Vaccine Use in Older Adults

Editorial Team — Thu, 15 May 2025 05:16:28 +0000

Following similar actions by France and the European Union, U.S. health authorities are urging a pause in the use of Valneva’s chikungunya vaccine, Ixchiq, in older adults.
The U.S. Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC) are recommending a temporary suspension of Ixchiq’s use in individuals aged 60 and older while investigations into serious adverse events (SAEs) continue on both sides of the Atlantic.
This joint advisory builds on the CDC’s April recommendation to administer the vaccine with caution in people aged 65 and above, according to a press release issued by Valneva on Monday. The European Medicines Agency (EMA) has also restricted the use of Ixchiq in older adults and those with weakened immune systems.

The EMA launched a safety review of the vaccine following reports of 17 serious adverse events, including two deaths, among recipients aged 62 to 89. The review began shortly after a similar move by French health authorities.
“As noted by the FDA and CDC, these adverse events may not be causally linked to the vaccine. However, a thorough evaluation is critical to ensure the safe use of Ixchiq, which has been administered in more than 40,000 doses globally,” Valneva stated in its latest response to the U.S. recommendation.
The company said it is coordinating with health authorities worldwide and expects structured reviews of post-marketing safety data in all regions where Ixchiq is approved.
Last month, Brazil became the first endemic country to grant marketing authorization for Ixchiq. The vaccine is also approved in the U.S., Canada, and the European Union.

Valneva emphasized its commitment to safety and collaboration with regulatory bodies, promising timely updates on all known serious adverse events. According to the company, most reported safety concerns have occurred in older individuals with pre-existing health conditions.
Despite the current scrutiny, Valneva is standing by Ixchiq. The vaccine was the first to receive FDA approval for chikungunya prevention in November 2023 and is intended for adults at increased risk of exposure to the virus, which is spread by mosquitoes in tropical regions.

FDA Clears First At-Home HPV and Cervical Cancer Screening Kit

Editorial Team — Tue, 13 May 2025 23:47:40 +0000

The U.S. Food and Drug Administration has approved the first at-home collection kit designed to allow women to screen themselves for human papillomavirus (HPV) and assess cervical cancer risk using a mailed-in sample. The device, developed by Teal Health and known as the Teal Wand, enables self-collection without a speculum and is available by prescription.
The Teal Wand uses a spongelike swab to collect a sample from within the vagina. Once mailed to a laboratory, the sample is analyzed using the same Pap smear test employed in clinical settings. According to a study conducted by Teal Health involving more than 600 participants, the samples collected with the Teal Wand were found to be as accurate as those collected by clinicians during in-office visits.

Participant feedback from the study indicated a high level of preference for at-home screening. Approximately 94% of participants said they would choose self-collection in the future, while 86% stated they believed they would be more likely to stay current with cervical cancer screenings if they could complete them at home.
Teal Health’s co-founder and CEO, Kara Egan, emphasized the importance of accessibility and convenience, particularly for women managing multiple responsibilities. “That’s why this FDA approval means so much; it’s not just about an innovative new product, it’s about finally giving women an option that makes sense for their lives—something that can be done quickly and comfortably at home,” she said in a statement.
This FDA decision follows prior approvals for HPV self-collection kits by BD and Roche in 2023. However, those earlier kits required sample collection within a healthcare clinic. In contrast, the Teal Wand is intended specifically for at-home use.
The product will initially be available through Teal Health’s website and telehealth platform starting in June, beginning in California, before becoming available nationwide. Patients will have virtual access to licensed medical providers who will prescribe the kit, evaluate the results, and offer support throughout the screening process.

The Teal Wand is intended for women aged 25 to 65 who are considered to be at average risk for cervical cancer. It previously received a breakthrough designation from the FDA.
Christine Conageski, M.D., principal investigator of the Teal Health trial and director of the Complex Dysplasia Clinic at the University of Colorado, noted that improving access is an essential step in addressing low screening rates. “The FDA approval of this at-home Teal Wand self-collection device is a critical step forward,” she said. “It offers an evidence-based way to expand access without compromising accuracy.”

Click Therapeutics Gains FDA Clearance for First Prescription Digital Migraine Prevention Tool

Editorial Team — Wed, 23 Apr 2025 16:53:14 +0000

Click Therapeutics has secured a notable regulatory milestone with the U.S. Food and Drug Administration granting authorization for CT-132, the agency’s first prescription digital therapeutic specifically designed to aid in the prevention of episodic migraines. This decision marks a significant advancement in the realm of digital health tools, particularly those intended to work in concert with conventional medication-based treatments.
The therapeutic, delivered via a smartphone application, underwent a randomized, sham-controlled clinical trial that demonstrated measurable improvements in reducing the number of monthly migraine days experienced by adult participants. In addition to achieving its primary goal, the study revealed that users of CT-132 also reported better outcomes in migraine-related quality of life and general health assessments.

The FDA’s clearance was obtained through the de novo pathway, a regulatory route designed for novel medical devices with no existing predicate. This designation not only affirms the program’s clinical validity but also provides a potential template for future advancements in software-based therapies. According to Click Therapeutics, this authorization opens the door for continued development of CT-132 as part of a broader treatment strategy that could include both digital and pharmaceutical elements.
Commenting on the development, Click’s CEO David Benshoof Klein highlighted that CT-132’s approval builds on the company’s prior successes across different areas of healthcare. “With this first-in-class FDA decision in the field of episodic migraine, our digital interventions have now achieved substantial clinical outcomes across psychiatry, cardiometabolic disorders, and now neurology,” Klein said in a statement.
The company has been expanding its digital therapeutics portfolio over recent years. In 2023, Click received regulatory clearance for a depression-focused application co-developed with pharmaceutical partner Otsuka. That same year, it also acquired a diabetes-related digital platform by purchasing the assets of the now-defunct Better Therapeutics, further broadening its scope.
Klein also emphasized the significance of the CT-132 approval as the company’s inaugural entry into neurological care and its first success addressing a pain-associated condition. “This authorization demonstrates the ability of our technology platform to yield tangible health benefits across a diverse set of treatment domains,” he added.

Click has indicated that CT-132 works by addressing brain hypersensitivity, specifically by adjusting the individual’s behavioral responses to external and internal triggers. In a separate analysis, the digital tool was shown to offer comparable benefits in individuals who were already undergoing treatment with CGRP inhibitors, medications commonly prescribed for migraines.
Most recently, the company announced the completion of a Series C financing round. Though financial details were not disclosed, the investment came entirely from Dassault Systèmes and its subsidiary, Medidata Solutions. The funding round also included a development collaboration agreement, suggesting continued momentum in Click’s pipeline expansion and innovation efforts.

GSK’s Blenrep Returns with U.K. Approval for Multiple Myeloma Combination Therapies

Editorial Team — Fri, 18 Apr 2025 20:51:02 +0000

GSK has received regulatory approval in the United Kingdom for two new combination therapies featuring its antibody-drug conjugate (ADC) Blenrep, marking a significant step in the drug’s return to the market following its previous withdrawal.
The Medicines and Healthcare products Regulatory Agency (MHRA) authorized the use of Blenrep (belantamab mafodotin) in combination with either bortezomib and dexamethasone, or pomalidomide and dexamethasone, for the treatment of relapsed or refractory multiple myeloma in adults who have received at least one prior line of therapy.
The decision is based on late-stage clinical trial data showing that these combinations improved progression-free survival and, in some cases, overall survival compared to standard-of-care regimens.

This marks a major milestone for Blenrep, which was pulled from global markets, including the U.S., in 2022 after a Phase 3 trial found it was not superior to existing therapies when used as a monotherapy. The MHRA approval supports its reintroduction in combination settings and signals renewed potential for the drug within GSK’s oncology pipeline.
Blenrep is part of the growing class of antibody-drug conjugates—engineered antibodies linked to cytotoxic agents that selectively target tumor cells while limiting off-target effects often associated with conventional chemotherapy.
GSK emphasized that the latest approvals position Blenrep for renewed growth, with regulatory reviews for the new combination regimens currently underway in 14 additional countries. The company also anticipates further approvals later this year.

The inclusion of established therapies such as bortezomib (a proteasome inhibitor), pomalidomide (an immunomodulatory agent), and the corticosteroid dexamethasone reflects a strategic approach to integrating Blenrep into existing treatment frameworks for multiple myeloma.

Vanda Challenges FDA Over Limits on Off-Label Drug Communication

Editorial Team — Wed, 16 Apr 2025 16:21:54 +0000

Vanda Pharmaceuticals is pushing back against the U.S. Food and Drug Administration (FDA), accusing the agency of stifling the dissemination of important medical information. The biotech company filed a lawsuit last week, aiming to secure the right to share data supporting off-label use of its sedative drug Hetlioz for treating jet lag.
The lawsuit, submitted on April 9 to the U.S. District Court for the Southern District of Texas, criticizes what Vanda describes as an overly restrictive regulatory framework. The company argues that the FDA is infringing on its First Amendment rights by limiting how pharmaceutical firms can inform healthcare professionals about alternative uses for approved medications, according to a report by Fierce Pharma.

Hetlioz, approved by the FDA in 2014 for non-24-hour sleep-wake disorder, later gained an additional approval to treat sleep disorders related to Smith-Magenis syndrome. Vanda now seeks to promote the drug for jet lag, an indication not currently authorized by the FDA.
While physicians are legally allowed to prescribe drugs for off-label uses, the FDA places tight controls on the type of information manufacturers can distribute regarding these unapproved applications. According to recent FDA guidance, companies must provide all necessary and accurate information to help healthcare providers evaluate the risks and benefits of such uses. However, the content must remain non-promotional, truthful, and not misleading. Communications must also clearly state that the use is not FDA-approved and disclose any known risks.
Vanda contends that these policies hinder innovation and delay the sharing of emerging clinical insights. In a press release, the company stated that the restrictions deter pharmaceutical companies from sharing valuable findings with physicians, fearing regulatory backlash. This, Vanda argues, ultimately harms patients by denying them access to potentially beneficial treatments.
In its legal filing, Vanda included clinical data demonstrating Hetlioz’s safety and effectiveness for jet lag patients, despite the lack of formal approval. The company is asking the court to affirm that this information is accurate and should be legally permissible to share.
This lawsuit adds to the growing tension between Vanda and the FDA. In 2019, the FDA declined to approve Hetlioz for jet lag, prompting Vanda to appeal. After receiving no response for over two years, the company sued the agency in 2022 for failing to meet required decision timelines.

The dispute doesn’t end there. In 2024, the FDA also rejected Vanda’s application for tradipitant, an NK-1R antagonist for gastroparesis. Vanda argued that the agency dismissed significant clinical evidence and missed its statutory 180-day decision deadline by over 185 days. The company took its case to then-FDA Commissioner Robert Califf in January, criticizing the agency for a lack of transparency and what it called a “culture of obfuscation and closemindedness.”
Shortly after Vanda’s letter, the FDA responded with its rationale for rejecting tradipitant and offered the opportunity for a hearing, signaling a small step toward dialogue amid the broader regulatory conflict.

Trump’s Proposed Pharmaceutical Tariffs May Potentially Drive Up Prices and Deepen Drug Shortages

Editorial Team — Fri, 11 Apr 2025 20:30:04 +0000

The U.S. may soon impose significant tariffs on pharmaceutical imports, according to President Donald Trump. At a recent National Republican Congressional Committee event, Trump said the initiative is intended to encourage the return of drug manufacturing to the United States. He also expressed concern over the comparatively lower drug prices in other countries. However, the proposed move has drawn scrutiny from patient advocates and supply chain analysts, who caution it may lead to higher medicine prices and aggravate current drug shortages.

Although Trump did not disclose specific tariff rates or timelines, the proposal follows an earlier announcement on April 2, in which pharmaceuticals were notably exempt. Medicines had also been excluded from tariffs during his first term. Trump emphasized that the U.S.’s limited capacity to produce essential antibiotics represents a critical issue, though experts argue tariffs may worsen this problem in the short term.

Generic drugs, which comprise approximately 90% of all prescriptions in the U.S., could be particularly affected. Many of these rely on ingredients sourced from China and India. According to Tom Kraus of the American Society of Health-System Pharmacists, the low profit margins on generics make them vulnerable to even small increases in production costs. A rise in prices for ingredients, he warns, could lead some manufacturers to halt production.

Drug shortages are already a growing concern. Data from the end of 2024 showed that 40 antibiotics were in short supply, including common treatments like amoxicillin and Bicillin. Sterile injectables used in hospitals, such as IV saline, dextrose, and chemotherapy agents, are also at risk, with shortages persisting due to production challenges and economic pressures.

Erin Fox, a drug shortage expert from the University of Utah Health, indicated that increased costs could prompt companies to exit the market, further weakening the pharmaceutical supply chain. Rena Conti of Boston University added that many generic drugs have only one or two ingredient suppliers, leaving the system highly susceptible to disruption.

The impact on branded drugs, which are typically more expensive and under patent protection, may be different. Financial analyst David Maris noted that while the cost of active ingredients in branded medications is relatively low, companies are still likely to pass on increased costs to consumers. This could result in higher insurance premiums and out-of-pocket expenses.

Group purchasing agreements, which lock in hospital drug prices for years at a time, may limit immediate price increases for some generics, according to Dr. Marta Wosińska of the Brookings Institution. However, these contracts do not guarantee supply, potentially leaving hospitals with limited access to critical medications.

Some industry observers, including Merith Basey of Patients for Affordable Drugs, argue that tariffs may worsen the already high drug prices in the U.S. Others, such as Umer Raffat of Evercore ISI, warn that price hikes could reignite political efforts to cap domestic drug prices using international benchmarks.

Despite the stated goal of encouraging domestic drug production, many experts remain skeptical about the effectiveness of tariffs in achieving that aim. Pharmaceutical supply chains are complex, and shifting manufacturing to the U.S. is a multi-year process. While companies like Eli Lilly have recently announced substantial investments in U.S.-based facilities, those projects are not expected to be operational for several years.

The industry is awaiting a potential Section 232 investigation by the U.S. Department of Commerce, which could enable tariffs based on national security considerations. However, some experts argue that broader systemic changes—including increased reimbursements and policy incentives—would be more effective in reshoring pharmaceutical manufacturing.

FDA Gives Reproxalap the Red Light For Dry Eye Disease

Editorial Team — Fri, 04 Apr 2025 19:59:40 +0000

The US Food and Drug Administration has sent a second detailed reaction letter to Aldeyra Therapeutics about its new drug application (NDA) for reproxalap. A pioneering small-molecule modulator of reactive aldehyde species (RASP), the medication is now undergoing evaluation for the treatment of dry eye disease.

The FDA endorsed Aldeyra’s updated NDA in November 2024, after the issuance of a comprehensive response letter requiring more evidence to substantiate effectiveness, particularly concerning ocular symptoms. This time around, the FDA said that the amended NDA did not prove effectiveness and requested at least one more controlled research, as reported by Aldeyra. The company’s press statement dated April 3 said that no production or safety concerns were detected.

Dry eye disease (DED), a prevalent cause for seeking ophthalmic treatment, is a complicated, multifactorial illness marked by tear film instability and persistent ocular surface irritation.

The primary objective of managing dry eye disease is to restore tear film equilibrium, which involves disrupting the cycle of inflammation. Although artificial tears are the cornerstone of treatment and provide symptomatic relief for DED, they fail to target the underlying disease processes. Current immunomodulators, such as cyclosporine and lifitegrast, often exhibit delayed onset and restricted tolerability, resulting in insufficient management of many patients’ DED.

Reproxalap targets RASP, namely malondialdehyde (MDA), which serve as upstream catalysts of inflammation by chemically altering cellular receptors and kinases.

The NDA for Reproxalap was substantiated by data from several studies, including the first Phase 3 TRANQUILITY study and the subsequent Dry Eye Chamber research after the initial NDA rejection. Collectively, these assessments examined both the immediate and long-term effectiveness of the medication in relieving ocular inflammation and patient-reported complaints.

The TRANQUILITY trial was a randomized, double-blind, vehicle-controlled Phase 2b/3 research study that included individuals with moderate to severe DED. Patients were subjected to a clinical setting intended to intensify dry eye symptoms and received either reproxalap 0.25% ophthalmic solution or a placebo.

The results indicated statistically significant reductions in ocular redness within hours after delivery, along with positive trends in ocular pain and tear stability. Nonetheless, while the sign goals were achieved, the FDA said that the symptom data were inadequate to warrant approval.

The agency demanded an extra carefully controlled experiment that specifically demonstrated effectiveness for ocular symptoms, resulting in the Dry Eye Chamber experiment. This Phase 3 experiment was developed in accordance with FDA instructions to directly evaluate symptom management during acute dry eye stress.

In both studies, including over 2,500 participants, reproxalap displayed a reliable safety profile. The predominant adverse event was moderate, temporary pain at the instillation site, with no discontinuations linked to therapy. No systemic safety concerns or novel signals were detected, confirming the drug’s appropriateness for long-term usage.

Aldeyra intends to disclose top-line findings from its current field study and chamber investigation in the second quarter of the year The firm announced its intention to resubmit its NDA soon this year.