UCB said it’s the first time an approved biologic demonstrated superiority over an IL-23 blocker in psoriatic arthritis. Yet analysts aren’t sure how commercially impactful the results will be.

Original title: “Bimzelx tops Skyrizi in head-to-head psoriatic arthritis trial”

Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for Biologics Evaluation and Research.

Original title: “Capricor Shares Rise as FDA Lifts Duchenne Cell Therapy Rejection”

NICE recommends non‑hormonal therapy for moderate to severe vasomotor symptoms

Original title: “NHS to offer fezolinetant for menopause‑related hot flushes and night‑sweats”

BE BOLD trial shows bimekizumab outperforms risankizumab at Week 16

Original title: “UCB reports superiority of bimekizumab in psoriatic arthritis study”

Xenon shares skyrocketed nearly 50% on results that handily beat Wall Street expectations. Elsewhere, Capricor got a decision date for a spurned cell therapy and AbbVie and Regeneron showcased obesity data.

Original title: “Xenon hits a ‘home run’ in epilepsy; FDA restarts review of Duchenne cell therapy”

The U.S. Food and Drug Administration today approved expanded use of Wellcovorin (leucovorin calcium) tablets for the treatment of cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1).

Original title: “FDA Approves First Treatment for Patients with Cerebral Folate Transport Deficiency ”

The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescription drug that the Trump administration touted from the Oval Office as a potential treatment for ... The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescription drug that the Trump administration touted from the Oval Office as a potential treatment for symptoms of autism last year. The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same prescrip… “This is going to be the first FDA-approved treatment for this ultra rare disease that is manifest with neurological complications, including seizures and developmental delay,” a senior health official said during in a press briefing. “And the approval is exciting because it was based on a systematic review of the literature,” they added. “It did not involve a randomized control trial, but the neurology review team did an exceptional job with their systematic review and was able to show that this was — had a high level of efficacy compared with what we see in the natural history of the cerebral folate transport deficiency disease.” According to the National Organization for Rare Disorders, there were less than 20 individuals who had been documented in medical literature with the disease in 2019.

Original title: “FDA approves Wellcovorin for ultra-rare disease. The Trump admin touted it as a treatment for autism”

Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according to analysts at Stifel.

Original title: “Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data”

Vertex Pharmaceuticals has a rolling biologics license application with the FDA for povetaciceptin IgA nephropathy. With new data from RAINIER, the biotech expects to complete its submission by the end of March.

Original title: “Vertex’s Nephropathy Asset Delivers ‘Strong’ Late-Stage Data, Clears Path to FDA”

Early data show favourable safety and sustained target engagement for oral EP4 receptor antagonist

Original title: “Kainova reports positive results for DT‑9081 in advanced solid tumours”

Study will assess whether microbiome therapy can protect gut diversity during transplantation

Original title: “EnteroBiotix completes enrolment for trial of EBX‑102‑02”

AstraZeneca Novartis AG Labcorp ... disease treatments.​ January 2026: FDA dropped certain manufacturing quality control requirements for cell and gene therapies targeting unmet needs in rare diseases, easing clinical development paths. January 2026: FDA implemented adaptive clinical trial allowances alongside prior Rare Disease Evidence Principles from September 2025, enabling approvals from single-arm studies. ​Japan: March 2026: Japan's MHLW granted orphan drug designation ... AstraZeneca Novartis AG Labcorp IQVIA ICON plc Parexel International (MA) Corporation Syneos Health Recent Developments: United States: February 2026: FDA launched a framework to accelerate individualized therapies for ultra-rare diseases, emphasizing disease-causing variant identification.​ February 2026: FDA introduced a "plausible mechanism pathway" to broaden gene-editing successes for rare disease treatments.​ January 2026: FDA dropped certain manufacturing quality control requirements for cell and gene therapies targeting unmet needs in rare diseases, easing clinical development path Unlock 360° Market Intelligence with DataM Subscription Services: https://www.datamintelligence.com/reports-subscription Conclusion: The Rare Disease Clinical Trials Market is poised for robust growth, fueled by rising prevalence of rare diseases, technological advancements, and favorable regulatory support. Biologics and gene therapies continue to dominate as preferred treatment modalities, while North America remains the leading regional market. Related Reports: Rare Disease Therapeutics Market Oncology Clinical Trials Market · Sai Kiran DataM Intelligence 4Market Research 877-441-4866 [email protected] Visit us on social media: LinkedIn X ... EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. The Global Rare Disease Clinical Trials Market is expected to reach at a CAGR of 9.39% during the forecast period 2026 to 2033. The Global Rare Disease Clinical Trials Market is rapidly expanding, driven by growing rare disease research, regulatory support and innovation, targeting significant unmet needs.”

Original title: “Rare Disease Clinical Trials Market is expected to reach US$ 30.13 Billion by 2033 | DataM Intelligence”

Company shares surged in after-market trading on results for a drug Vertex acquired in a multibillion-dollar deal and sees as having “best-in-class potential” in a competitive field.

Original title: “Vertex kidney disease drug hits mark in late-stage study”

The result “pulls the rug” out from under giredestrant, according to one analyst, sparking another share selloff following disappointing obesity data last week.

Original title: “Roche’s prized breast cancer pill fails closely watched study”

AbbVie’s foray into the obesity space is successful so far, analysts agree, as amylin analog ABBV-295 elicited as much as 9.73% weight loss at 13 weeks in the multiple ascending dose portion of a Phase 1 trial.

Original title: “AbbVie’s Amylin Candidate ‘Competitive’ in Early-Stage Trial”

Hansoh’s olatorepatide achieved 19% weight loss at 48 weeks in a Phase 3 trial in China, handing partner Regeneron a glimmer of hope for a pipeline in desperate need of reinvigoration.

Original title: “Regeneron’s Weight Loss Partner Hansoh Delivers Much-Needed Phase 3 Win in China”

Roche’s shares tumbled nearly 5% on news that a key pillar in the five-pronged clinical plan for the breast cancer asset giredestrant was unsuccessful.

Original title: “Roche Breast Cancer Prospect Fails First-Line Test, Shares Fall”

Azetukalner, a Kv7 potassium channel opener, reduced the frequency of focal onset seizures by a placebo-adjusted rate of 42.7%. Xenon Pharmaceuticals believes this is the highest such efficacy “observed in a pivotal epilepsy study,” CEO Ian Mortimer said Monday.

Original title: “Xenon To Seek Approval of First-in-Class Epilepsy Drug After Exceeding Phase 3 Expectations”

Ipsen will withdraw Tazverik’s follicular lymphoma and epithelioid sarcoma indications as emerging data point to an elevated safety risk in patients undergoing treatment.

Original title: “Ipsen Pulls Cancer Drug Amid Risk of Secondary Malignancies”

The FDA's cancer advisors will discuss AstraZeneca’s application for the oral SERD camizestrant in breast cancer and the AKT inhibitor Truqap in prostate cancer.

Original title: “Two AstraZeneca Drugs To Be Scrutinized in First FDA Cancer Advisory Panel in 9 Months”