Each month we scan the regulatory landscape to bring you the latest developments shaping pharmaceutical and biotechnology innovation. In this roundup you’ll find key publications that caught our attention, plus a curated summary of the most important regulatory news. Our goal is to highlight what matters, cut through the noise, and keep you up to date on changes that could impact drug development and market access in Europe and the US.
News
The EMA recommended Aqneursa to treat Niemann-Pick type C disease
The EMA has recommended granting marketing authorisation for Aqneursa (levacetylleucine) to treat neurological symptoms of Niemann-Pick type C (NPC) disease in patients aged 6 years and older weighing at least 20 kg. Aqneursa can be used with miglustat or as monotherapy when miglustat is not tolerated. NPC is a rare, progressive, fatal genetic disorder affecting lipid metabolism, primarily in children, with no curative treatments. Levacetylleucine, a modified amino acid, improves neurological function by correcting energy metabolism and increasing adenosine triphosphate production in cerebellar cells. The recommendation is based on a randomised, double-blind, placebo-controlled, two period crossover study in 60 patients, measuring neurological function via the Scale for the Assessment and Rating of Ataxia (SARA) scale. Aqneursa significantly improved SARA scores compared with placebo, including in patients not on miglustat. Patients switching from Aqneursa to placebo experienced symptom worsening, supporting its efficacy in NPC.
The EMA recommends a new injection for easier prevention of HIV-1
The EMA has recommended marketing authorisation for Yeytuo (lenacapavir) for human immunodeficiency virus type 1 (HIV-1) pre-exposure prophylaxis (PrEP) in adults and adolescents at high risk, alongside safer sex practices. Yeytuo is administered via subcutaneous injection twice a year, improving uptake and adherence compared with daily oral PrEP.Lenacapavir, a first-in-class agent, binds HIV-1 capsid proteins, disrupting viral replication and preventing infection. The recommendation is based on two randomised, double-blind, active-controlled trials. In PURPOSE 1, cisgender women aged 16–24 received Yeytuo (n=2134) or Truvada (n=1068); no new infections occurred in the Yeytuo group versus 16 in Truvada. In PURPOSE 2, men and gender-diverse persons received Yeytuo (n=2179) or Truvada (n=1086); two infections occurred in the Yeytuo group versus nine in Truvada. Participants on Yeytuo showed higher adherence. This accelerated approval addresses unmet needs in HIV prevention globally.
The EMA published its report on the real-world evidence framework
The third annual report highlights progress in integrating Real-World Evidence (RWE) into regulatory decision-making under the European Medicines Regulatory Network (EMRN) 2028 strategy. The EMA coordinated 59 studies via DARWIN EU®, framework contracts and in-house studies, with 33 completed and 26 ongoing; a 47.5% increase from the prior year. Of the 90 assessed research topics, 78% were deemed feasible for RWE studies, facilitated by DARWIN EU’s expansion to 30 data partners across 16 countries, which covers approximately 180 million patients.
Studies addressed drug utilisation (42%), safety (24%) and disease epidemiology (24%), supporting regulatory and public health decision-makers, including EMA committees, ECDC, Health Technology Assessment bodies and the European Commission. Topics included suicidality risks with doxycycline and glucagon-like peptide 1agonists, medicine shortages, and mpox vaccine effectiveness. Median study duration for DARWIN EU® was 4 months, enabling faster integration into regulatory timelines. Efforts also improved data quality, analysis methods and transparency, including the launch of HMA-EMA RWD catalogues.
The MHRA approves elinzanetant to treat moderate to severe vasomotor symptoms for menopause
The MHRA has become the first regulator worldwide to approve elinzanetant (Lynkuet) for the treatment of moderate to severe vasomotor symptoms (hot flushes) associated with menopause. Menopause-related drops in oestrogen cause overactive brain cells that disrupt temperature regulation, leading to hot flushes and night sweats. Elinzanetant is a non-hormonal oral medication that calms these signals, helping restore temperature control and potentially improving sleep. Approval is based on the OASIS clinical trials involving over 1400 women aged 40–65 years across multiple countries. Participants taking a daily 120 mg capsule of Lynkuet experienced a significant reduction in the number and intensity of hot flushes and night sweats over 26–52 weeks compared with placebo.
The MHRA approves Ekterly to treat hereditary angioedema
Alongside the FDA and EMA, the MHRA also approved Ekterly (sebetralstat) to treat hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. Sebetralstat is the first and only MHRA-approved oral, on-demand treatment for HAE. It is taken as soon as symptoms begin, giving patients the ability to treat the attacks as soon as they begin, without the need for injections.
The MHRA approved tofersen to treat rare inherited form of motor neurone disease
The MHRA approved tofersen (Qalsody) for adults with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, a rare inherited form of motor neurone disease (MND). ALS progressively damages nerve cells in the brain and spinal cord, leading to muscle weakness, including muscles needed for breathing and swallowing. Tofersen works by reducing levels of the toxic protein produced by the SOD1 gene, protecting nerve cells from damage. Approval was granted via the International Recognition Procedure. The treatment is administered by lumbar puncture at regular intervals by a healthcare professional.
The MHRA approved mirvetuximab to treat ovarian, fallopian tube or primary peritoneal cancer
The MHRA approved mirvetuximabsoravtansine (Elahere) for adults with ovarian, fallopian tube or primary peritoneal cancer whose tumours are Folate Receptor alpha (FRα) positive and who have not responded to platinum-based chemotherapy after one to three prior treatments. Approval was granted via the International Recognition Procedure.Mirvetuximabsoravtansine is a monoclonal antibody linked to a cancer drug (DM4), which binds to FRα on cancer cells, enters the cell, and releases DM4 to kill the cancer cells. It is administered intravenously, with dosing based on body weight and cycles determined by the doctor. Approval was supported by a study of 453 adults with advanced platinum-resistant FRα-positive cancers. Patients receiving mirvetuximabsoravtansine had a median progression-free survival of 5.6 months versus 4 months with standard chemotherapy, and overall survival of 16.5 months versus 12.8 months, demonstrating improved outcomes over standard treatment.
The MHRA approved nogapendekin alfa inbakicept (Anktiva) for adults with BCG-unresponsive non-muscle invasive bladder cancer
The MHRA approved nogapendekin alfa inbakicept (Anktiva) for adults with BCG(Bacillus Calmette-Guérin)-unresponsive non-muscle invasive bladder cancer, where tumours remain confined to the bladder lining. BCG, the standard early-stage immunotherapy, is delivered directly into the bladder, but some patients do not respond, leaving limited treatment options. Anktiva was approved through the International Recognition Procedure, which allows the MHRA to consider decisions from trusted regulatory partners to benefit UK patients. The treatment is administered by mixing nogapendekin alfa inbakicept with BCG in a liquid, which is then delivered into the bladder via a catheter inserted through the urethra.
The FDA publishes over 200 complete response letters
The FDA has published over 200 complete response letters (CRLs) issued between 2020 and 2024, enhancing transparency in drug and biological product approvals. CRLs are sent to Sponsors when applications cannot be approved as submitted, typically due to safety, efficacy, manufacturing or bioequivalence concerns, and include recommendations for addressing these deficiencies. Historically, CRLs were not publicly available, leading Sponsors to omit up to 85% of the FDA’s safety and efficacy concerns when announcing non-approvals. Publication of these letters now allows the public and industry to gain insight into the FDA’s decision-making and learn from common application deficiencies. The initial batch, linked to since-approved applications, is accessible via openFDA, with redactions for trade secrets and confidential information. The FDA plans to release additional CRLs and continues exploring ways to increase transparency and share lessons learned from non-approvals.
ESMO welcomes the European Commission’s Life Sciences Strategy
The European Society of Medical Oncology (ESMO) welcomes the European Commission’s Life Sciences Strategy, particularly its commitment to simplify EU regulations on medical devices and in vitro diagnostics (IVDR) by the end of 2025. This follows ESMO’s joint call with 51 leading European cancer centres, highlighting the threat posed by the current IVDR application to oncology research and patient access to innovative treatments. ESMO praises the Commission’s commitment to reform legislation to enable clinical research and innovation, rather than obstruct it. Measures to improve the implementation of the Clinical Trials Regulation, especially for multi-country trials, are recognised as critical but require adequate resources. With over 40,000 members worldwide, many in Europe, ESMO is ready to continue supporting EU institutions with evidence-based expertise. The organisation envisions a thriving European life sciences ecosystem by 2030, fostering innovation and delivering breakthroughs that improve the lives of millions of cancer patients.
News: New regulation for cutting-edge cancer treatments and personalised gene therapies to be made where patients are treated
UK patients gain faster access to personalised medicines under new legislation came into force on 23 July. The MHRA’s world-first regulation, known as The Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations 2025, allows breakthrough therapies, including cell and gene therapies, tissue-engineered treatments, 3D printed products, blood products and medicinal gases, to be prepared in small or individual batches at hospitals, clinics, or near patients’ homes. This means cancer patients could have immune cells modified to fight their tumour returned within days, and children with rare genetic disorders could receive therapies with only minutes of shelf life. Previously, treatments like CAR-T (chimeric antigen receptor T-cell) therapy required distant specialised manufacturing, causing delays or missed opportunities.
The legislation establishes regulated protocols for on-site or mobile manufacturing, cutting waiting times, freeing NHS beds and supporting community or home-based care. The MHRA has published guidance and provides scientific advice to ensure safe implementation, strengthening the UK’s leadership in decentralised, point-of-care medicine manufacturing.
The FDA published a draft guidance on approaches to assessment of overall survival in oncology clinical trials
This guidance provides recommendations for Sponsors on assessing overall survival (OS) in randomised oncology clinical trials supporting marketing approval of drugs and biologics, with a focus on OS as a pre-specified safety endpoint. Based on discussions from the July 2023 FDA–AACR–ASA public workshop, it addresses statistical and design considerations, particularly when OS is not the primary endpoint. The guidance emphasises that OS and other time-to-event endpoints are difficult to interpret in single-arm trials due to the absence of a randomised control arm, reinforcing the importance of randomised designs for demonstrating treatment effects.
Publications that caught our eye...
Thirty years of Europe’s centralized procedure for approving medicines. Nature Medicine.
Totality of the Evidence: Optimizing Dosage Selection Strategies in Oncology. Journal of Clinical Oncology.
Acquired resistance in cancer: towards targeted therapeutic strategies. Nature Reviews Cancer.
Surrogate end points in oncology: aligning drug development incentives and patient needs. Nature Reviews Clinical Oncology.
Reply to ‘Surrogate end points in oncology: aligning drug development incentives and patient needs’. Nature Reviews Clinical Oncology.
Multiplicity Control in Clinical Trials. Clinical Trials Workshop.
Therapy for Stage IV non-small cell lung cancer with driver alterations: ASCO Living Guideline, version 2025.1. Journal of Clinical Oncology.
