Regulatory concerns for Novo plant
Following a recent inspection, the FDA has classified a Catalent facility in Indiana owned by Novo Nordisk as Official Action Indicated. According to the FDA website, the label indicates that a “facility is in an unacceptable state of compliance. The facility may have been issued a Form FDA-483 or FDA-4056 at the conclusion of the inspection.”
Scholar Rock, which is a customer of the site, says it received news of the action from Novo Nordisk. A statement says: “We continue to work closely with Novo Nordisk, and we have requested a Type A meeting with FDA to discuss next steps for resubmitting our BLA. We plan to share more information during our third quarter 2025 business update call in November.”
Media reports have also suggested that Regeneron may use the site.
AstraZeneca signs agreement with US government
AstraZeneca says it has reached an agreement with the US government under the Inflation Reduction Act “to lower the cost of prescription medicines for American patients while preserving America’s cutting-edge biopharmaceutical innovation.” A range of measures have been agreed to give Americans access to AstraZeneca medicines at “prices that are equalized with those available in wealthy countries.” Direct-to-consumer sales will be provided to eligible patients and the company will also be participating in the TrumpRx.gov direct purchasing platform.
The terms of the agreement, including financial details and the specific products involved, were not disclosed. The company says it has also reached an agreement with the Department of Commerce to delay Section 232 tariffs for three years.
Epilepsy improvements
UCB has published the final analysis from an open-label extension study evaluating the long-term safety and effectiveness of Fintepla (fenfluramine) in patients with Lennox-Gastaut syndrome. Over a median treatment period of one year, patients experienced a median 31 percent reduction in the frequency of seizures associated with falls (drop seizures), with improvements seen as early as month 2 and sustained through the study’s end. Adults showed a 40 percent median reduction, while pediatric patients achieved a 27.6 percent reduction. Nearly 60 percent of patients were rated by caregivers and investigators as showing overall improvement.
Caregivers also reported better social interactions, reduced feelings of stigma, and lower anxiety and depression levels. Importantly, no new safety signals emerged, with the most common treatment-emergent adverse events being decreased appetite, fatigue, and nasopharyngitis.
Kelly G Knupp, Associate Professor of Pediatrics and Neurology at the CU Anschutz School of Medicine, and co-author of the paper, said, “These data reinforce the long-term safety and tolerability of fenfluramine in children and adults living with Lennox-Gastaut syndrome, a condition with a significant unmet medical need. The sustained reduction in seizure frequency, coupled with the meaningful improvements in quality of life for both patients and their families, underscores the importance of addressing not just the clinical but also the emotional and social challenges faced by these families every day.”
Conditional marketing authorization for radioconjugate
The EMA has confirmed that Collectar Biosciences’ investigational therapeutic iopofosine I-131 is eligible for submission of a Conditional Marketing Authorization as a treatment for patients with Waldenström macroglobulinemia (WM), a B-cell malignancy characterized by bone marrow infiltration with clonal lymphoplasmacytic cells that produce a monoclonal immunoglobulin M that remains incurable with available treatments.
Iopofosine is a novel radioconjugate using the company’s phospholipid ether platform. CEO James Caruso said, “We are thrilled to take this important step toward bringing iopofosine I-131 to patients in Europe living with WM. With PRIME designation already in hand and feedback from the SAWP, we are rapidly proceeding toward a potential European approval and commercial availability in 2027.”
New guidance on pluripotent stem cell-derived therapies
The International Society for Stem Cell Research (ISSCR) has released a new guidance document: Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies. The publication provides a structured framework for advancing human pluripotent stem cell (PSC)-derived therapies from research to clinical and commercial stages, covering PSC line selection, qualification of raw and ancillary materials, manufacturing, regulatory strategy, preclinical testing, and clinical trial design.
The guidance is designed to be jurisdictionally neutral, offering adaptable principles that can align with regional regulatory requirements. Emphasis is placed on maintaining consistent quality control, risk management, and traceability across the development pipeline. ISSCR says it will update the document annually to reflect any new scientific, technological, and regulatory advances.
Gene therapy success in profound genetic hearing loss
Regeneron reported new results from a pivotal trial of its investigational gene therapy DB-OTO for children with congenital hearing loss caused by otoferlin (OTOF) gene variants. The data show that 11 of 12 participants experienced clinically meaningful improvements in hearing, with three achieving normal hearing levels. Improvements were typically observed within weeks of treatment and were sustained or continued to improve in participants followed for up to 72 weeks.
The trial involves a single intracochlear infusion of DB-OTO, which delivers a working copy of the OTOF gene using a dual adeno-associated virus vector. Hearing assessments by pure tone audiometry and auditory brainstem response confirmed restoration of hearing function in most participants. Among those assessed for speech perception, all demonstrated measurable gains, including the ability to recognize words without visual cues.
Regeneron plans to file for US regulatory approval later in 2025, following discussions with the FDA. The therapy has received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations.
Amoxicillin analysis
A report compiled by Portugal-based research consultancy New Angle has highlighted Amoxicillin as a central example of the challenges facing off-patent antibiotics in Europe. The report notes that availability has fluctuated in recent years, with shortages driven by rising production costs, API concentration, and reduced manufacturer profitability. It also shows how 27 of 125 Amoxicillin products (21.6 percent) were withdrawn from the market, with shortages affecting 69 products. The analysis links these shortages to broader systemic issues, including production fragility and policy-driven price suppression. Amoxicillin, according to the report, exemplifies how economic and regulatory pressures are destabilizing Europe’s antibiotic supply chains.
FDA approval for Arcutis
The FDA has approved Arcutis Biotherapeutics’ Zoryve (roflumilast) cream for the treatment of mild to moderate atopic dermatitis in pediatric patients. In a pediatric study of 652 children, 39.4 percent reached a 75 percent reduction in disease severity. This marks the sixth FDA approval in the Zoryve portfolio within just over three years. Arcutis CEO Frank Watanabe said, “Zoryve cream was intentionally formulated to address the critical unmet need in atopic dermatitis care, and it provides a safe, effective alternative to steroid treatments, targeting the underlying inflammation that drives disease activity. Notably, Zoryve does not include ingredients known to compromise the skin barrier, which is especially important for young children with atopic dermatitis.”
Labelling update from Abbvie
The FDA has approved Abbvie’s updated indication for RINVOQ (upadacitinib) for adults with moderately to severely active ulcerative colitis and Crohn’s disease. The revision allows use of the drug after at least one approved systemic therapy when TNF blockers are clinically inadvisable, expanding beyond the prior requirement of prior TNF blocker failure or intolerance. The updated label maintains existing safety warnings, including risks of serious infections, malignancies, cardiovascular events, and thrombosis.
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