Intriguing science
While blood brain barrier is a key protection for the brain, emerging evidence suggests that specific components of the BBB, the mucins, are key to protect brain against effects of aging. Is it the new target to prevent diseases correlated with neurological aging?
European Medicine Agency (EMA)
The EMA’s Committee for Medicinal Products for Human Use (CHMP) had their monthly meeting from 24-27 February 2025. Here are the key takeaways:
- Deqsiga (human normal immunoglobulin, Takeda) was granted a recommendation for marketing authorization for replacement therapy in people with primary or secondary immunodeficiencies and immunomodulation such as primary immune thrombocytopenia.
- Lynozyfic (linvoseltamab, Regeneron) was recommended for conditional marketing authorization for the treatment of relapsed and refractory multiple myeloma.
- Vyjuvek (beremagene geperpavec, Krystal Biotech) received a positive opinion for the treatment of wounds in patients of all ages with dystrophic epidermolysis bullosa.
- Trabectedin Accord (trabectedin, Accord Healthcare) received a positive opinion for the treatment of advanced soft tissue sarcoma and of relapsed platinum-sensitive ovarian cancer.
- Kaftrio (ivacaftor/tezacaftor/elexacaftor, Vertex) and Kalydeco (ivacaftor, Vertex) received a recommendation for a label expansion to include their use in combination in patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- Ixchiq (chikungunya vaccine (live), Valneva) received a positive opinion for active immunisation of adolescents from 12 years of age.
- Fabhalta (iptacopan, Novartis) received a positive opinion for an indication extension for the treatment of adult patients with complement 3 glomerulopathy.
- Leqembi (lecanemab, Biogen and Eisai) received another positive opinion for marketing authorization following a re-examination.
US Food and Drug Administration (FDA)
Ctexli (chenodiol, Mirum Pharmaceuticals) was approved for the treatment of adults with cerebrotendinous xanthomatosis. Mirum Pharmaceuticals press release.
Odactra (allergen extract immunotherapy, ALK) was approved for use in young children with house dust mite (HDM) allergy. ALK 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 February 17 and March 2, 2025:
- Supernus Pharmaceuticals announced that the Phase 2b study of SPN-820 in adults with treatment-resistant depression (TRD) did not meet its primary endpoint. Supernus press release.
- Emalex Biosciences announced that its Phase 3 study of ecopipam in subjects with Tourette syndrome met its primary and secondary endpoints. Emalex press release.
- AstraZeneca shared positive data from the Phase 3 study of camizestrant in patients with hormone receptor (HR)-positive, HER2-negative advanced breast cancer whose tumours have an emergent ESR1 mutation. AstraZeneca press release.
- Recce Pharmaceuticals announced that the Phase 2 trial of Recce 327 for the treatment of Acute Bacterial Skin and Skin Structure Infections (ABSSSI) met its primary and secondary endpoints. Recce press release [pdf].
- Bristol Myers Squibb reported positive results from the Phase 3 study of Opdivo (nivolumab) in combination with platinum-doublet chemotherapy as a neoadjuvant treatment for adult patients with resectable (tumors ≥ 4 cm or node positive) non-small cell lung cancer (NSCLC). BMS press release.
- Sellas Life Sciences shared positive data from the Phase 2a trial of SLS009 (tambiciclib) in relapsed/refractory Diffuse Large B-Cell Lymphoma (r/r DLBCL). Sellas press release.
- Johnson & Johnson reported positive data from the Phase 3 study of Tremfya (guselkumab) in adults with moderately to severely active ulcerative colitis (UC). J&J press release.
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