The European Medicines Agency's Committee for Advanced Therapies (CAT) has recommended two new advanced therapy medicinal products (ATMPs) for EU approval during the March–May 2026 period, according to the CAT quarterly highlights published on 24 June 2026. Adstiladrin (nadofaragene firadenovec) received a positive opinion for treating adult patients with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, while Itvisma (onasemnogene abeparvovec) was recommended for spinal muscular atrophy in patients aged 2 years and older. The European Commission granted marketing authorisation for Adstiladrin on 28 May 2026, and Itvisma is pending Commission decision.
The CAT quarterly report, covering March to May 2026, details the committee's activities on ATMPs. Adstiladrin, a gene therapy, was assessed by CAT in March 2026, leading to a positive draft opinion. The Committee for Medicinal Products for Human Use (CHMP) subsequently adopted a positive opinion, recommending conditional marketing authorisation. Itvisma, another gene therapy, received a positive CAT draft opinion in April 2026, followed by CHMP endorsement for full marketing authorisation. No extensions of indication for already authorised ATMPs were reported in this period.
The report also provides statistical data on product-related activities. Since 2009, 51 marketing authorisation applications (MAAs) for ATMPs have been submitted, with 34 positive opinions (including the two new ones) and 7 negative opinions. In 2026, two MAAs received positive opinions (Adstiladrin and Itvisma), and no new submissions or withdrawals occurred. For variations (Type II) of authorised ATMPs, 15 positive opinions were issued in 2026, contributing to a total of 313 since 2009. Scientific recommendations on advanced therapy classification saw 20 submissions and 14 adoptions in 2026, with cumulative totals of 737 submissions and 727 adoptions.
The approvals benefit patients with rare or hard-to-treat conditions: bladder cancer patients now have a gene therapy option after BCG failure, and older SMA patients gain access to a treatment previously limited to younger children. For the pharmaceutical industry, the positive opinions signal regulatory pathways for gene therapies, though conditional authorisation for Adstiladrin means post-marketing data obligations. EU healthcare systems will need to budget for high-cost therapies, potentially straining national health budgets. The CAT's continued activity reinforces the EU's role in fostering advanced therapies, but the low number of new submissions in 2026 (zero so far) suggests a cautious pipeline. No institutional follow-up is specified beyond the Commission's final decisions.