Drug News: 30 June 2025

Intriguing Science

A recent article in Nature attracted our attention, AI models used in the medical field to help clinicians may worsen with time. This is due to the fact that they are trained using data they impacted. E.g. the AI suggest to initiate an intervention to avoid sepsis, the clinician does it and therefore sepsis is avoided. In the next training, the AI may find that there is not so much of risk of sepsis associated with that set of parameters anymore. And therefore, update the model on wrong assumptions. Read more, it’s fascinating!

European Medicine Agency (EMA)

The EMA’s Committee for Medicinal Products for Human Use (CHMP) held its monthly meeting from June 16 to 19, 2025. Here are the key takeaways:

1. Austedo (deutetrabenazine, Teva) received a positive opinion for the treatment of adults with moderate-to-severe tardive dyskinesia.
2. Imreplys (sargramostim, Partner Therapeutics) received a positive opinion for marketing authorisation under exceptional circumstances for the treatment of patients with haematopoietic acute radiation syndrome.
3. Ogsiveo (nirogacestat, SpringWorks Therapeutics) received a positive opinion for the treatment of adults with progressing desmoid tumours.
4. Rezdiffra (resmetirom, Madrigal Pharmaceuticals) received a positive recommendation for conditional marketing authorization for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH).
5. Zemcelpro (dorocubicel / unexpanded umbilical cord cells, Cordex Biologics) received a positive recommendation for conditional marketing authorization to treat patients with haematological malignancies.
6. The CHMP adopted positive opinions for six biosimilar medicines:

• Mynzepli (aflibercept, Advanz Pharma), Afiveg (aflibercept, Stada Arzneimittel), Vgenfli (aflibercept, Zaklady Farmaceutyczne Polpharma), and Eiyzey (aflibercept, Zaklady Farmaceutyczne Polpharma), for the treatment of age-related macular degeneration and visual impairment.
• Usymro (Ustekinumab, Elc Group), for the treatment of plaque psoriasis, arthritis psoriatic and Crohn’s Disease.
• Vivlipeg (pegfilgrastim, Biosimilar Collaborations), to reduce the duration of neutropenia and the incidence of febrile neutropenia after cytotoxic chemotherapy. CHMP meeting highlights.

US Food and Drug Administration (FDA)

1. Monjuvi (tafasitamab-cxix, Incyte) was approved in combination with lenalidomide and rituximab for adults with relapsed or refractory follicular lymphoma (FL). Incyte press release.
2. Datroway (datopotamab deruxtecan-dlnk, Daiichi Sankyo) received FDA accelerated approval for adults with locally advanced or metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC). FDA press release.
3. Benlysta (belimumab, GSK) was approved for subcutaneous injection in patients five years of age and older with active lupus nephritis (LN) who are receiving standard therapy. GSK press release.
4. Dupixent (dupilumab, Sanofi) was approved for the treatment of adult patients with bullous pemphigoid (BP). Sanofi press release.
5. Andembry (garadacimab-gxii, CSL) was approved to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. CSL press release.
6. Yeztugo (lenacapavir, Gilead Sciences) was approved as pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35kg. Gilead press release.

On the safety side, the FDA is investigating two deaths in patients treated with Elevidys (Sarepta Therapeutics) for Duchenne muscular dystrophy. The agency stated the deaths appear treatment-related and will assess the need for additional regulatory action. FDA press release.

New data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between June 16 and 29, 2025:

1. AbbVie announced that the Phase 3 trial evaluating venetoclax in combination with azacitidine in the treatment of newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) did not meet the primary endpoint. AbbVie press release.
2. Nuvalent shared positive results from the Phase 1/2 study of zidesamtinib in TKI (tyrosine kinase inhibitor) pretreated patients with advanced ROS1-positive non-small cell lung cancer (NSCLC). Nuvalent press release.
3. Compass Pathways announced that the Phase 3 trial evaluating COMP360 for treatment-resistant depression (TRD) met its primary endpoint. Compass press release.
4. Scholar Rock reported positive results from the Phase 2 trial of apitegromab in combination with tirzepatide to preserve lean mass during tirzepatide-induced weight loss. Scholar Rock press release.
5. Altimmune shared positive results from the Phase 2b trial of pemvidutide in metabolic dysfunction-associated steatohepatitis (MASH). Altimmune press release.
6. Nektar Therapeutics announced that the Phase 2b study of rezpegaldesleukin in patients with moderate-to-severe atopic dermatitis met both its primary and secondary endpoints. Nektar press release.
7. Lyell Immunopharma reported positive data from the Phase 1/2 trial of LYL314 for the treatment of aggressive large B-cell Lymphoma. Lyell Immunopharma press release.