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 published a draft guideline on the inclusion of pregnant and breastfeeding women in clinical trials
The EMA has published a draft guideline for public consultation aimed at improving the inclusion of pregnant and breastfeeding women in clinical trials. Developed through the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, the guideline signals a paradigm shift in how medicines are studied for these populations. It recommends that inclusion be considered for all medicines intended for people who can become pregnant and sets out conditions to protect participants and their offspring. Currently, fewer than 0.4% of EU trials include pregnant people, and only 0.1% include lactating individuals, leading to critical data gaps. As a result, product leaflets often lack safety and efficacy information for use in pregnancy and breastfeeding, potentially compromising care. The guideline promotes early dialogue between developers and regulators and encourages both pre- and post-authorisation research to generate robust evidence for safer, more informed treatment decisions during pregnancy and lactation.
First treatment against liver scarring caused by a type of ‘fatty liver disease’
The EMA has recommended conditional marketing authorisation for Rezdiffra (resmetirom) to treat adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced liver fibrosis, alongside diet and exercise. MASH, a serious liver condition linked to fat accumulation and inflammation, can progress to cirrhosis or cancer. Rezdiffra is the first potential EU-approved treatment for this condition. Its active substance, resmetirom, activates liver-specific thyroid hormone receptor β (THR‑β), reducing liver fat, inflammation, and fibrosis.
Approval is based on a pivotal trial of 917 patients. After 12 months, 30% of those receiving 100 mg and 26% receiving 80 mg achieved MASH resolution without fibrosis worsening, versus 10% on placebo. Fibrosis improvement without MASH worsening was seen in 29% (100 mg), 27% (80 mg) and 17% (placebo). Supportive studies and ongoing trials are required to confirm long-term efficacy.
New stem cell therapy to treat patients with blood cancers
The EMA has recommended conditional approval of Zemcelpro (dorocubicel/unexpanded umbilical cord cells) for adults with haematological malignancies requiring allogeneic stem cell transplant but lacking a suitable donor. Zemcelpro combines standard and expanded umbilical cord blood cells to enhance cell counts and improve engraftment. In two single-arm studies (25 patients), 84% achieved neutrophil engraftment within 20 days and 68% platelet engraftment within 40 days. Common side effects include cytopenias, infections, hypertension, and graft-versus-host disease. Supported through the EMA’s PRIority MEdicines scheme, Zemcelpro addresses an unmet need in transplant-eligible patients without donor options. Benefits were deemed to outweigh potential risks.
The MHRA approved serplulimab (Hetronifly) to treat extensive-stage small cell lung cancer
On 20 June 2025, the MHRA approved serplulimab (Hetronifly) for the treatment of adults with previously untreated extensive-stage small cell lung cancer that has spread within the lungs or to other parts of the body. Small cell lung cancer is an aggressive form of lung cancer, accounting for 10–15% of cases, and is often diagnosed at an advanced stage. Serplulimab is a monoclonal antibody that targets the programmed cell death protein 1 (PD-1) receptor, which cancer cells use to evade immune detection. By blocking PD-1, serplulimab enhances the immune system’s ability to identify and destroy cancer cells. Hetronifly was submitted and approved under the International Recognition Procedure.
A patient in the UK has become the first in Europe to receive leniolisib (Joenja) for Activated PI3-Kinase Delta Syndrome
A patient in the UK received leniolisib (Joenja) on the National Health Service (NHS) for Activated PI3-Kinase Delta Syndrome (APDS), a rare, inherited immune disorder. The treatment was administered at Addenbrooke’s Hospital following over a decade of Cambridge-led research. APDS is caused by overactive PI3Kδ signalling, leading to recurrent infections, immune dysfunction and increased lymphoma risk. Leniolisib, a selective oral PI3Kδ inhibitor taken twice daily, targets the underlying disease mechanism, offering a safer alternative to antibiotics, immunoglobulin therapy or bone marrow transplants. Already approved in the US in 2023, it received National Institute for Health and Care Excellence approval in 2025, making the NHS the first European healthcare system to offer it. The treatment is expected to significantly improve quality of life for APDS patients.
The FDA removed the Risk Evaluation and Mitigation Strategies for six approved autologous chimeric antigen receptor T-cell therapies
The FDA has removed the Risk Evaluation and Mitigation Strategies (REMS) for Abecma, Breyanzi, Carvykti, Kymriah, Tecartus and Yescarta – six approved autologous chimeric antigen receptor T-cell (CAR T) therapies used to treat blood cancers such as multiple myeloma, leukaemia and lymphoma. REMS, a safety programme for high-risk drugs, is no longer deemed necessary as safety risks like cytokine release syndrome and neurological toxicities are now well-managed through labelling and clinical practice. The change eliminates certification requirements for hospitals and the need for on-site tocilisumab access, reducing burdens on providers and improving patient access. FDA officials emphasised that physicians now have greater experience managing CAR T-related toxicities, and ongoing safety monitoring remains a priority.
Publications that caught our eye...
Efficiency must not compromise trustworthiness in rating certainty and formulating recommendations in AI era. The British Medical Journal.
