With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.

Original title: “BioMarin Pauses Dosing, Enrollment in 2 Voxzogo Studies After Safety Signal”

Our team is currently finalizing ... gene therapy targeting GNE Myopathy and we anticipate that a Phase I clinical trial may begin within the next 12-months. Solve GNE remains focused on turning scientific progress into real momentum for GNE Myopathy patients and their families. Our commitment to funding additional research will help build a stronger path toward future treatment options for this rare disease... Our team is currently finalizing the IND application for the first gene therapy targeting GNE Myopathy and we anticipate that a Phase I clinical trial may begin within the next 12-months. Solve GNE remains focused on turning scientific progress into real momentum for GNE Myopathy patients and their families. Our commitment to funding additional research will help build a stronger path toward future treatment options for this rare disease. Solve GNE highlights new research, expanded funding efforts, and progress toward a Phase I clinical trial for GNE myopathy.... About Solve GNE Solve GNE is a nonprofit foundation dedicated to advancing research and treatment development for GNE myopathy. Through fundraising and scientific partnerships, Solve GNE works to accelerate progress toward therapies for patients and families affected by this rare muscle disease. LOS ANGELES, March 16, 2026 (GLOBE NEWSWIRE) -- Solve GNE, a nonprofit foundation dedicated to accelerating treatments for GNE myopathy, has announced major progress across its research, fundraising, and regulatory efforts. To date, the non-profit has raised more than $6.5 million in support of programs aimed at moving clinically viable therapies closer to approval.

Original title: “Solve GNE Highlights New Research, Expands Funding Efforts, and Advances Toward a Phase I Clinical Trial for GNE Myopathy”

Study to assess oral therapy for patients with inadequate response to TNF inhibitors

Original title: “Elevara begins phase 2b trial of ELV001 in rheumatoid arthritis”

Structure Therapeutics’ oral obesity drug elicited a placebo-adjusted weight loss of 16.3% at 44 weeks in a Phase 2 trial. The biotech is planning to launch a late-stage program for the drug later this year.

Original title: “Structure Climbs on ‘Highly Competitive’ Profile for Weight Loss Pill”

Phase 3 trial delivers significant skin clearance and symptom improvement

Original title: “Lebrikizumab shows strong results for children with atopic dermatitis”

Sana Biotechnology is looking to start clinical development for its type 1 diabetes therapy SC451 this year.

Original title: “Sana’s Cell Therapy Sustains Insulin Production Through 14 Months in a T1D Patient”

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO's investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.

Original title: “Denali’s Hunter Syndrome Candidate in the Spotlight After REGENXBIO Rejection”

The trial clearance for Shanghai-based Unixell is another step forward for the rapidly advancing Chinese biotech ecosystem, which has invested heavily in cell and gene therapy technology.

Original title: “With FDA go ahead, a China biotech notches a first in cell therapy testing”

The approval represents a win in what’s been a difficult regulatory environment of late for vaccine makers, and could boost uptake of a shot that’s posted flattening sales after an initially strong launch.

Original title: “GSK’s RSV vaccine wins broader FDA clearance”

EMA annual report 2025The Management Board adopted EMA’s annual report for 2025, marking another strong year for medicines regulation in the European Union. EMA delivered 104…

Original title: “EMA Management Board: highlights of March 2026 meeting”

CVMP opinions on veterinary medicinal productsThe Committee adopted a positive opinion for a marketing authorisation for AviGate S. Infantis, for active immunisation of…

Original title: “Meeting highlights from the Committee for Veterinary Medicinal Products (CVMP) 10-12 March 2026”

Oncology dominates due to the high ... orphan oncology drugs. Neurology and genetic disorders are growing areas as advancements in gene therapy and personalized medicine create new opportunities for rare disease treatment. Hematology, metabolic disorders, and immunology are also key segments with increasing clinical research ... Oncology dominates due to the high prevalence of rare cancers and strong investment in developing orphan oncology drugs. Neurology and genetic disorders are growing areas as advancements in gene therapy and personalized medicine create new opportunities for rare disease treatment. Hematology, metabolic disorders, and immunology are also key segments with increasing clinical research activities. Genomic, precision medicine, and cell & gene therapies are rapidly advancing, offering targeted treatments for rare diseases. Regenerative medicine and omics-based approaches are emerging as future growth drivers. By Region North America - 40% Share North America leads the market due to advanced clinical research infrastructure, regulatory support for orphan drugs, high R&D investments, and strong collaboration between pharmaceutical companies, research institutes, and patient advocacy groups in the U.S. Press release - DataM Intelligence 4Market Research LLP - Rare Disease Clinical Trials Market to Reach US$ 30.13 Billion by 2033, Growing at 9.39% CAGR Driven by Gene Therapy, Precision Medicine, and Biomarker?Based Innovations with Key Players Pfizer Inc. Novartis AG Roche Holding AG and Emerging Innovators T - published on openPR.com ✅ 2025: Growing healthcare investments, regulatory support for orphan drugs, and increasing access to patient registries in China, Japan, and India boosted the development and execution of rare disease clinical trials. Recent Key Developments - Product & Technology Innovation ✅ 2025: Gene Therapy & Personalized Medicine Trials: Development of targeted gene therapies and individualized treatment approaches expanded opportunities for rare disease clinical research.

Original title: “Rare Disease Clinical Trials Market to Reach US$ 30.13 Billion by 2033, Growing at 9.39% CAGR Driven by Gene Therapy, Precision Medicine, and Biomarker‐Based Innovations with Key Players Pfizer Inc. Novartis AG Roche Holding AG and Emerging Innovators T”

PRAC warns about known risk of aseptic meningitis with chikungunya vaccine IxchiqUpdate to product information recommended to reflect recent evidenceEMA’s safety committee (…, Agenda Agenda of the PRAC meeting 9-12 March 2026Draft Reference…, PRAC statistics: March 2026 , PRAC statistics: March 2026

Original title: “Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 9 - 12 March 2026”

An Ultragenyx gene therapy has scored a win at the midway | An Ultragenyx gene therapy has scored a win at the midway point of its phase 3 trial, successfully reducing ammonia levels in patients with a rare urea cycle disorder. One-time treatment with DTX301 led to 18% lower ammonia levels compared to placebo at 36 weeks, hitting one of the trial's two key endpoints. That gene therapy snub, combined with a pair of phase 3 fails for its brittle bone disease antibody, prompted Ultragenyx to lay off 10% of its workforce last month. Now, the company also has a class-action lawsuit stemming from its communications around the brittle bone disease data to contend with. Ultragenyx urea cycle disorders rare diseases Cell & Gene Therapy Clinical Trial Results clinical trial data Clinical Data Biotech Thirty-six weeks after one-time treatment with the AAV-based gene therapy DTX301, patients’ ammonia levels were 18% lower than placebo, a statistically significant result that met one of the trial’s co-primary endpoints, Ultragenyx reported March 12. After meeting the measure for change in 24-hour plasma ammonia levels, the trial still must meet another primary endpoint—the percent of patients who achieve a response as measured by discontinuation or reduction in baseline disease management. A positive OTC deficiency readout would provide a morale boost for Ultragenyx, as the rare disease specialist has struggled with some setbacks as of late. The company recently resubmitted its application for FDA approval of its Sanfilippo syndrome type A gene therapy, which the agency initially rejected back in July 2025 due to manufacturing concerns.

Original title: “Ultragenyx gene therapy hits co-primary endpoint in phase 3 rare disease study, teeing up potential win”

Regulator authorises deuruxolitinib for adults with extensive hair loss

Original title: “MHRA approves new treatment for severe alopecia areata”

Study aims to advance treatment options for severe peripheral arterial disease

Original title: “Aplagon doses first patient in phase 2a trial of APAC”

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular dystrophy.

Original title: “BridgeBio Builds Case for Early 2027 Launch of Dystrophy Drug”

It is estimated that more than ... a clinical spectrum of disease severity. In the late-onset form of the disease, elevated ammonia can lead to significant medical issues for patients. Approved therapies, which must be taken multiple times a day for the patient's entire life, do not eliminate the risk of future metabolic crises. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare ... It is estimated that more than 10,000 people are affected by OTC deficiency in commercially accessible geographies, of whom approximately 80% are classified as late-onset and represent a clinical spectrum of disease severity. In the late-onset form of the disease, elevated ammonia can lead to significant medical issues for patients. Approved therapies, which must be taken multiple times a day for the patient's entire life, do not eliminate the risk of future metabolic crises. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. Statistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeks Clinically important changes observed in... (NASDAQ: RARE) announced positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301-treated patients (n=18) demonstrated a statistically significant and clinically meaningful 18% (p=0.018) reduction in 24-hour plasma ammonia (AUC0-24) compared to placebo (n=19) and maintained average ammonia AUC0-24 in the normal range through Week 36. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics.

Original title: “Ultragenyx Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency”

Months after promising help for “hundreds of thousands of kids,” the FDA approved the decades-old medicine for a disease documented in less than 50 people.

Original title: “FDA clears repurposed GSK drug for ultra-rare brain disease instead of autism”