Paltusotine

The EMA's drug advisory panel issued its latest basket of recommendations and rejections on Friday, throwing its support behind six drugs, whilst declining to back two treatments. The most notable nod went to Moderna for mCombriax (mRNA-1083), a combination mRNA vaccine designed to protect adults 50 years and older against both seasonal flu and COVID-19. The shot's support from European regulators comes shortly after the FDA initially declined to review a US application for the standalone flu portion of the jab — mRNA-1010 — before reversing course and promising a decision by August 5. With the positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in-hand, Moderna is now awaiting a final marketing authorisation decision on mCombriax from the European Commission. Good news for Crinetics, NovartisJust five months after Crinetics Pharmaceuticals won US clearance for its once-daily oral acromegaly treatment, it now has CHMP backing for Palsonify (paltusotine) in the EU.The nonpeptide somatostatin receptor type 2 (SST2) agonist is supported by data from two Phase III pivotal trials, PATHFNDR-1 and PATHFNDR-2, which respectively enrolled previously treated and treatment-naïve adults with acromegaly, a rare hormonal disorder caused by the the production of excess growth hormone. In both studies, a significantly higher proportion of participants receiving Palsonify maintained IGF-1 levels ≤1 times the upper limit of normal compared with those on placebo, alongside significant reductions in acromegaly-associated symptoms.A FirstWord analysis pegged Crinetics as a likely takeout target this year, given its positive US launch of Palsonify (see – Vital Signs: The seller's marketplace heading into JPM).On a similar timeline as Crinetics, Novartis also bagged CHMP backing after a September FDA approval; the Swiss pharma got a positive recommendation for Rhapsido (remibrutinib) to treat chronic spontaneous urticaria (CSU).In the late-stage REMIX-1 and REMIX-2 studies, Rhapsido outperformed placebo on standard measures of itch, hive counts and overall disease activity at 12 weeks, with significantly more treated patients than placebo recipients reporting control of symptoms as early as the second week.In the US, the BTK inhibitor is indicated for adults who remain symptomatic despite taking antihistamines. As a twice-daily pill, it offers patients with CSU an oral alternative to existing injectable therapies.Nods for Ipsen, X4Also earning a thumbs up from CHMP was Ipsen and its paediatric brain cancer treatment, Ojemda (tovorafenib). The conditional marketing recommendation covers its use as monotherapy in patients aged 6 months and older with low-grade glioma harbouring a BRAF fusion or rearrangement or BRAF V600 mutation and who have progressed after one or more prior systemic therapies. Ipsen licensed the pan-RAF kinase inhibitor from Day One Biopharmaceuticals in 2024, after it received FDA approval in a similar setting. Rounding out the positive opinions was X4 Pharmaceuticals' Xolremdi (mavorixafor), which got a nod from CHMP "under exceptional circumstances" to treat patients aged 12 and up with WHIM syndrome, an ultra-rare immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction; The disease is named for its four classic manifestations: warts, hypogammaglobulinaemia, infections and myelokathexis.Despite winning FDA approval in 2024 for the CXCR4 antagonist, X4 has struggled to remain afloat. The company in 2025 underwent two separate rounds of layoffs to stretch its cash runway to a topline readout of the Phase III 4WARD study evaluating Xolremdi in chronic neutropenia, expected next half. Negative opinions for Acadia, VandaOne of Friday's two rejections was not a surprise. Acadia Pharmaceuticals disclosed earlier this month that it expected to receive a negative opinion for trofinetide in Rett syndrome, a rare neurodevelopmental condition. Acadia has said it will seek a re-examination of its application for the IGF-1 analogue, which is already approved in the US and Canada under the brand name Daybue.The other thumbs-down, however, went to an atypical antipsychotic that has been cleared in the US to treat schizophrenia since 2009. More recently, Vanda Pharmaceutical's Fanapt (iloperidone) got a nod from the FDA in 2024 for the acute treatment of manic or mixed episodes associated with bipolar I disorder in adults. In its rejection of iloperidone in both disease settings, CHMP wrote that the drug was associated with pronounced QT prolongation, "which is not outweighed by the benefits of treatment." Vanda may ask for re-examination of the opinion within 15 days.

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has recommended 12 medicines for approval at its February 2026 meeting, including a first-in-class combined mRNA vaccine targeting both seasonal influenza and COVID-19. At the forefront, the CHMP recommended granting a marketing authorization for mCombriax, a combined influenza and COVID-19 mRNA vaccine developed by Moderna. Designed for adults aged 50 and older, mCombriax combines two messenger RNA constructs into a single formulation intended to simplify annual respiratory virus immunisation. The recommendation now goes to the European Commission for final approval. According to World Health Organization data, Europe has recorded more than 281 million COVID-19 cases since the pandemic began, while seasonal influenza alone causes up to 50 million symptomatic cases annually in the European Economic Area (EEA). In oncology, the CHMP recommended a conditional marketing authorisation for Ojemda (tovorafenib) for patients aged six months and older with pediatric low-grade glioma, a non-cancerous brain tumor. The once-weekly oral targeted therapy – developed by Day One Biopharmaceuticals, with ex-US rights licensed to Ipsen SA – aims to expand treatment options beyond surgery and conventional chemotherapy, which can have modest benefit and significant toxicity in young patients. The committee also adopted a positive opinion for Onerji, a levodopa/carbidopa product developed by Tanabe Pharma GmbH, for adults with advanced Parkinson’s disease. In endocrinology, Recordati Rare Diseases-partnered paltusotine, marketed as Palsonify by Crinetics Pharmaceuticals, received a positive opinion for acromegaly, a rare disorder of excess growth hormone. Immunology saw a positive opinion for Rhapsido (remibrutinib) from Novartis for chronic spontaneous urticaria, a long-term itchy rash. For rare inherited immune dysfunction, the CHMP adopted an opinion under exceptional circumstances for Xolremdi (mavorixafor) from X4 Pharmaceuticals, intended for patients aged 12 and older with WHIM syndrome, an ultra-rare condition marked by recurrent infections and increased malignancy risk. The committee also endorsed six biosimilar medicines. Among them was Poherdy, a pertuzumab biosimilar developed by Shanghai Henlius Biotech and partnered with Organon for global ex-China commercialization. If approved, Poherdy would be the first pertuzumab biosimilar endorsed by the CHMP, broadening competition to Roche’s reference product Perjeta in the EU. Poherdy was approved by the US FDA in November 2025. Additional biosimilars recommended include insulin lispro Bysumlog and insulin aspart Dazparda (both from Gan & Lee Pharmaceuticals, and marketed by Sandoz); etanercept Fubelv (by Biosimilar Collaborations Ireland); tocilizumab Tuyory (developed by Gedeon Richter in collaboration with Mochida Pharmaceutical); and teriparatide Zandoriah (by Cinnagen Co. Unipessoal Lda). Final marketing authorizations are subject to European Commission decisions, typically issued within two to three months following CHMP positive opinions.