Drug News: 16 June 2025

Intriguing Science

Science fiction is now! The team of Wairagkar, University of California implemented 256 electrodes in the brain of a patient with ALS. After a learning phase, using previous recording of the patient, the implemented brain computer interface could reproduce the patients words and other speech features: intonation, pitch and emphasis.

Read more in Nature.

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European Medicine Agency (EMA)

Since the last newsletter, the CHMP held its monthly meeting from May 19 to 22, 2025. Here are the key takeaways:

1. Aucatzyl (obecabtagene autoleucel, Autolus) received a positive recommendation for conditional marketing authorization for the treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukaemia.
2. Blenrep (belantamab mafodotin, GSK) received a positive opinion for the treatment of relapsed or refractory multiple myeloma.
3. Ezmekly (mirdametinib, SpringWorks Therapeutics) received a positive recommendation for conditional marketing authorization for the treatment of plexiform neurofibromas in pediatric and adult patients.
4. Itovebi (inavolisib, Roche) received a positive opinionfor the treatment of adults with PIK3CA-mutated, oestrogen receptor (ER)-positive, HER2-negative locally advanced and metastatic breast cancer.
5. Maapliv (amino acids, Recordati) received a positive opinion under exceptional circumstances for the treatment of maple syrup urine disease (MSUD) in patients from birth.
6. Riulvy (tegomil fumarate, Neuraxpharm Pharmaceuticals) received a positive opinion for the treatment of adults and children from 13 years of age with relapsing remitting multiple sclerosis.
7. Atropine sulfate FGK (atropine, FGK) received a negative opinion for treating myopia in children aged three years and older.
8. Kinselby (resminostat, 4SC) received a negative opinion for the treatment of patients with advanced stage mycosis fungoides and Sezary syndrome.
9. The CHMP adopted positive opinions for three biosimilars : Bomyntra (denosumab, Fresenius Kabi), Conexxence (denosumab, Fresenius Kabi) and Rolcya (denosumab, Sandoz).

CHMP meeting highlights.

The  Pharmacovigilance organ (the PRAC) held its meeting from June 2 to 5, 2025. Here are the key takeaways:

For eye condition NAION, a very rare side effect of semaglutide medicines:

• PRAC has completed its review of semaglutide-containing medicines following concerns about a potential increased risk of non-arteritic anterior ischemic optic neuropathy (NAION).
• The committee recommends updating the product information for semaglutide medicines to include NAION as a side effect with a frequency of “very rare.”
• If NAION is diagnosed, treatment with semaglutide should be discontinued.

Risk of encephalitis with varicella vaccines:

• PRAC is currently reviewing the known risk of encephalitis with two varicella (chickenpox) vaccines, Varilrix and Varivax, following a report of a fatal outcome after vaccination with Varilrix.
• While the investigation is ongoing, both vaccines can continue to be used in line with the approved product information. PRAC meeting highlights.

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US Food and Drug Administration (FDA)

1. mRESVIA (mRNA-1345 (RSV vaccine), Moderna) received a label expansion approval for the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 18-59 years of age who are at increased risk for disease. Moderna press release.
2. Enflonsia (clesrovimab-cfor, Merck) was approved for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in neonates (newborns) and infants who are born during or entering their first RSV season. Merck press release.
3. Mavyret (glecaprevir/pibrentasvir, AbbVie) received a label expansion approval for the treatment of adults and pediatric patients three years and older with acute or chronic hepatitis C virus (HCV) infection without cirrhosis or with compensated cirrhosis. AbbVie press release.
4. Keytruda (pembrolizumab, Merck) was approved as a single-agent neoadjuvant treatment for adults with resecable, locally advanced head and neck squamous cell carcinoma (HNSCC) expressing PD-L1. Merck press release.
5. Zusduri (mitomycin, UroGen Pharma) intravesical solution was approved for adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). UroGen Pharma press release.
6. Nubeqa (darolutamide, Bayer) was approved for metastatic castration-sensitive prostate cancer (mCSPC). FDA press release.
7. Ibtrozi (taletrectinib, Nuvation Bio) was approved for the treatment of adult patients with locally advanced or metastatic ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC). Nuvation Bio press release.
8. Tryptyr (acoltremon ophthalmic solution, Alcon) was approved for the treatment of signs and symptoms of Dry Eye Disease (DED). Alcon press release.
9. Khindivi (hydrocortisone oral solution, Eton Pharmaceuticals) was approved as a replacement therapy in pediatric patients five years of age and older with adrenocortical insufficiency. Eton Pharmaceuticals press release.
10. Yutrepia (Treprostinil, Liquidia) was approved for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) to improve exercise ability. Liquidia press release.
11. Zoryve (roflumilast, Arcutis Biotherapeutics) was approved for the treatment of plaque psoriasis of the scalp and body in adult and pediatric patients 12 years of age and older. Arcutis Biotherapeutics press release.
12. Susvimo (ranibizumab injection, Genentech) was approved for the treatment of diabetic retinopathy (DR). Genentech press release.
13. Nucala (mepolizumab, GSK) was approved as an add-on maintenance treatment for adult patients with inadequately controlled COPD and an eosinophilic phenotype. GSK press release.
14. Nuvaxovid (COVID-19 vaccine, Novavax) was approved for active immunization to prevent COVID-19 in adults 65 years and older and individuals 12-64 years who have at least one underlying risk factor. Novavax press release.
15. mNEXSPIKE (mRNA-1283, Moderna), a new vaccine against COVID-19, was approved for use in all adults aged 65 and older and individuals aged 12-64 years with at least one underlying risk factor. Moderna press release.

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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 May 190 and June 15, 2025:

1. Vera Therapeutics announced that the Phase 3 trial of atacicept for the treatment of immunoglobulin A nephropathy (IgAN) in adults met its primary endpoint. Vera press release.
2. Gilead announced the FDA has placed clinical hold on HIV treatment trials of GS-1720 and/or GS-4182. The clinical hold is due to the identification of a safety signal of decreases in CD4+T-cell (CD4) and absolute lymphocyte counts in a subset of participants receiving the combination of GS-1720 and GS-4182. Gilead press release.
3. Merck shared positive results from two Phase 3 trials evaluating enlicitide decanoate as add-on treatment of adults with hyperlipidemia. Merck press release.
4. Otsuka Pharmaceutical presented positive results from the Phase 3 study evaluating sibeprenlimab, for the treatment of immunoglobulin A nephropathy (IgAN) in adults. Otsuka press release.
5. REGENXBIO announced positive data from the Phase 1/2 trial of RGX-202 for Duchenne muscular dystrophy. REGENXBIO press release.
6. Vigil Neuroscience announced that the Phase 2 trial evaluating iluzanebart for the potential treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) will be discontinued after iluzanebart showed no beneficial effects on biomarker or clinical efficacy endpoints. Vigil Neuroscience press release.
7. Sagimet Biosciences reported that the Phase 3 trial of denifanstat for the treatment of acne vulgaris met all primary and secondary endpoints. Sagimet Biosciences press release.
8. Regeneron announced positive results from the Phase 2 trial investigating combinations of semaglutide and trevogrumab in reducing weight with limited reduction of lean mass vs semaglutide only. Regeneron press release.
9. Insmed announced that the Phase 2b study of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary arterial hypertension met its primary and all secondary efficacy endpoints. Insmed press release.
10. Sobi and Apellis Pharmaceuticals presented positive data from the Phase 3 study of Aspaveli (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Sobi press release.
11. Valneva announced positive from its Phase 2 trial evaluating Ixchiq (chikungunya vaccine) in children. Valneva press release.
12. Rocket Pharmaceuticals announced that a patient in the Phase 2 trial of RP-A501 for Danon disease experienced an unexpected Serious Adverse Event (SAE) and subsequently died from an acute systemic infection. The SAE involved clinical complications related to a capillary leak syndrome. The FDA ordered the halt of the trial. Rocket Pharmaceuticals press release.
13. Roche reported positive results from the Phase 2 study of fenebrutinib in patients with relapsing multiple sclerosis (RMS). Roche press release.
14. GSK and Spero Therapeutics announced that the Phase III trial evaluating tebipenem HBr for the treatment of complicated urinary tract infections (cUTIs), including pyelonephritis, met its primary endpoint and will stop early for efficacy following an Independent Data Monitoring Committee review. GSK press release.
15. Veru shared positive results from the Phase 2b study of enobosarm to promote fat loss and lean mass loss in older patients (≥60 years) receiving Wegovy (semaglutide, Novo Nordisk) for chronic weight management. Veru press release.
16. Sanofi and Regeneron announced that itepekimab met the primary endpoint in one of two COPD Phase 3 studies. Sanofi press release.
17. Prothena reported that the Phase 3 trial evaluating birtamimab in patients with AL amyloidosis did not meet its primary endpoint. Prothena press release.
18. Tourmaline Bio shared positive results from its Phase 2 trial evaluating pacibekitug in patients with elevated high-sensitivity C-reactive protein (hs-CRP). Tourmaline Bio press release.
19. Apnimed announced that the Phase 3 trial AD109 (aroxybutynin /atomoxetine) in adults with mild, moderate, and severe obstructive sleep apnea (OSA) met its primary endpoint. Apnimed press release.
20. Ionis Pharmaceuticals reported positive results from the Phase 3 study of olezarsen in people with moderate hypertriglyceridemia with or at risk for atherosclerotic cardiovascular disease (ASCVD). Ionis press release.
21. Gilead Sciences announced that the Phase 3 study of Trodelvy (sacituzumab govitecan-hziy) in patients with first-line metastatic triple-negative breast cancer (mTNBC) who are not candidates for PD-1/PD-L1 inhibitors met its primary endpoint. Gilead press release.