Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.

Original title: “REGENXBIO, Novartis, Dyne near FDA submissions in muscular dystrophies”

CONTRAINDICATIONS Existence of ... disease History of transplantation and receiving immunosuppressant agents · WARNINGS AND PRECAUTIONS Patients exhibiting the following events should be closely monitored and may require dose reduction or discontinuation of therapy: Depression and Suicide: Monitor closely for symptoms and need for treatment... CONTRAINDICATIONS Existence of or history of severe depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons or any inactive ingredients Moderate or severe hepatic impairment History or presence of active serious or untreated autoimmune disease History of transplantation and receiving immunosuppressant agents · WARNINGS AND PRECAUTIONS Patients exhibiting the following events should be closely monitored and may require dose reduction or discontinuation of therapy: Depression and Suicide: Monitor closely for symptoms and need for treatment. INDICATION BESREMi® is indicated for the treatment of adults with polycythemia vera. IMPORTANT SAFETY INFORMATION WARNING: RISK OF SERIOUS DISORDERS · Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. Hepatotoxicity: Monitor liver enzymes and hepatic function at baseline and during treatment. Reduce dose or discontinue depending on severity. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Discontinue if severe renal impairment develops. Dental and Periodontal Toxicity: Advise patients on good oral hygiene and to have regular dental examinations. Dermatologic Toxicity: Consider discontinuing if clinically significant dermatologic toxicity. PharmaEssentia will also present an integrated analysis of the SURPASS-ET and Phase 2b EXCEED-ET (NCT05482971) trials. The analysis includes 182 patients across both studies and evaluates the consistency of clinical benefit across treatment lines, including treatment-naïve and previously treated populations, as well as across molecular subtypes and ethnicities.

Original title: “PharmaEssentia to Present New Clinical Data Supporting the Use of Ropeginterferon Alfa-2b in Essential Thrombocythemia at ASCO and EHA 2026 - BioSpace”

New findings presented at AAN 2025 showed that treatment with satralizumab was associated with a reduction in annualized relapse rate compared with placebo over 48 weeks in patients with MOGAD. The primary end point of the METEOROID study was time from randomization to the first adjudicated MOGAD relapse during the double-blind treatment period, as assessed by an independent clinical adjudication committee. Secondary end points included annualized relapse rate, the annualized rate of active lesions on MRI across the optic nerves, brain, and spinal cord, as well as the proportion of patients requiring rescue therapy, and the annualized rate of inpatient hospitalizations. No new safety signals were reported with satralizumab,, and the safety profile was consistent with established data from more than a decade of satralizumab, clinical trial and post-approval experience in aquaporin-4 immunoglobulin (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder. Adverse events (AEs) ≥5% and more commonly observed in patients receiving satralizumab, vs. placebo included injection-related reactions (16%), influenza (9%), arthralgia (9%), back pain (9%), sinusitis (7%) and diarrhea (6%). There were low rates of AEs leading to temporary treatment interruption with satralizumab, (6%) and placebo (5%). There was one fatality not related to treatment, and none of the serious AEs were considered related to treatment. The company noted that no new safety signals were identified with satralizumab, and its safety profile was reported to be consistent with established data from more than a decade of clinical trial and post-approval experience in aquaporin-4 immunoglobulin seropositive neuromyelitis optica spectrum disorder. Genentech noted that data from the METEOROID trial are planned for submission to regulatory authorities worldwide.1 · Findings showed that 87% of patients treated with satralizumab were relapse free at 48 weeks compared with 67% of those receiving placebo, with onset of response observed as early as 8 weeks (P = .0025). Notably, treatment effects were generally consistent across subgroups, including age, sex, race, and background therapy use.

Original title: “Satralizumab Meets Primary End Point in Phase 3 METEOROID Study in MOGAD | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

High-level results have led the companies to scrap the mid-stage trial, though Denali plans to continue independently running a separate experiment focused on a certain subset of Parkinson’s patients.

Original title: “Parkinson’s drug from Biogen, Denali comes up short”

Treatment with the TROP2 ADC sac-TMT led to a 70% objective response rate and progression-free survival was “significantly improved” as compared to placebo—the second positive readout for the asset this week.

Original title: “Kelun-Merck validate TROP2-Keytruda pairing in lung cancer with improved survival”

CVMP opinions on veterinary medicinal productsThe Committee adopted a positive opinion for a marketing authorisation for Nobivac NXT HC, for the active immunisation of …

Original title: “Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 19-21 May 2026”

Biogen and Denali’s Parkinson’s disease drug failed to significantly slow disease progression in a Phase 2b study, missing both primary and secondary endpoints.

Original title: “Biogen, Denali pull plug on Parkinson’s program after mid-stage flop”

Eight new medicines recommended for approvalEMA’s human medicines committee (CHMP) recommended eight medicines for approval at its May 2026 meeting.Jascayd (nerandomilast)…, CHMP statistics Key figures from the May 2026 CHMP meeting are represented in the graphic below.  , CHMP statistics:…, Positive recommendations on new medicines,

Original title: “Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 18-21 May 2026”

EMA has recommended granting a marketing authorisation in the European Union (EU) for Jascayd (nerandomilast) to treat adults with idiopathic pulmonary fibrosis (IPF) or…

Original title: “New medicine for two types of pulmonary fibrosis”

The success came two days after a Phase 3 setback for another experimental drug and helps improve the position of Voxzogo amid increased competition.

Original title: “BioMarin notches win in study that could expand use of top-selling medicine”

Participants who stayed on the drug lost around one-quarter of their body weight, potentially setting a new benchmark for rivals like Novo Nordisk.

Original title: “Lilly’s triple-acting obesity drug hits goal in Phase 3 trial”

Lilly met analysts’ sky-high expectations with 28.3% weight loss over 80 weeks for the triple hormone receptor agonist retatrutide in a highly anticipated readout on Thursday.

Original title: “Lilly’s triple agonist shows bariatric surgery–like weight loss results”

EMA’s office is closed on 25 May, to mark Whit Monday. The office reopens 08:30 on Tuesday, 26 May.The product emergency hotline is available outside working hours and on…

Original title: “EMA closed on Whit Monday, 25 May”

After being hit by safety issues and subpar results in another trial, BioMarin’s Phase 3 test of Voxzogo for a rare skeletal disorder called hypochondroplasia showed efficacy “solidly above” what the drug has shown for achondroplasia, which causes dwarfism.

Original title: “BioMarin bounces back as Voxzogo performs ‘bigger than expected’ in new indication”