Update as of 3 March 2026This news was updated to accurately reflect the number of medicines with a new active substance recommended for marketing authorisation.In 2022, EMA…

Original title: “Human medicines: highlights of 2022”

Company targets new approach to treating rheumatoid arthritis and systemic sclerosis

Original title: “Quell launches chill phase 1/2 trial for novel cartreg therapy”

The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.

Original title: “Intellia’s CRISPR Gene Therapy Free To Move Forward After FDA Fully Lifts Hold”

In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker levels or neurodevelopmental outcomes.

Original title: “REGENXBIO’s Hunter Rejection Hinged on Inadequate Controls, Surrogate Endpoint”

The FDA believes that an experimental therapy for Huntington’s disease from UniQure wasn't providing benefit for patients, a senior official told STAT. UniQure has accused the agency of reversing its stance on AMT-130, but hopes to settle on the parameters of a new trial. The Food and Drug Administration has concluded that an experimental treatment for Huntington’s disease developed by UniQure was not providing benefit for patients based on existing clinical data, a senior FDA official told STAT, explaining the agency’s decision to block the company from submitting a marketing application. Agency reviewers “are not convinced there’s any therapeutic benefit of the product,” the official said, speaking on condition of anonymity.

Original title: “FDA official: Agency ‘not convinced’ UniQure Huntington’s therapy has benefit”

Apazunersen, an investigational intrathecal therapy, is being evaluated in the phase 3 Aspire trial for pediatric Angelman syndrome, with study completion anticipated in the second half of 2026. Apazunersen, an investigational intrathecal therapy, is being evaluated in the phase 3 Aspire trial for pediatric Angelman syndrome, with study completion anticipated in the second half of 2026. ... Welcome to NeurologyLive®'s Clinical Trial in Focus. Every month, an ongoing clinical trial in the landscape of neurology is featured, highlighting the design of the study, the targeted patient population, the enrollment criteria, the primary and secondary end points, and its potential implications for clinical care. This month’s spotlight is the global phase 3 Aspire study (NCT06617429) assessing apazunersen (Ultragenyx Pharmaceutical), an investigational antisense oligonucleotide therapy, in pediatric patients with Angelman syndrome (AS).1 Additional exclusions included known hypersensitivity to apazunersen or its excipients, medical conditions or laboratory abnormalities that could compromise safety or interfere with study assessments, pregnancy or breastfeeding, prior exposure to investigational treatments, including gene therapy or antisense oligonucleotides, or concurrent enrollment in another interventional trial. This region, discovered by Scott Dindot, PhD, is believed to be the structural start site and important for its regulation," Kim Goodspeed, MD, MS, senior medical director of global clinical development at Ultragenyx, told NeurologyLive. "Precise targeting of this site resulted in robust and efficient repression of the UBE3A-antisense transcript and reactivation of the paternal copy of UBE3A throughout the [central nervous system] of cynomolgus macaques." Enrollment for the Aspire study began in December 2024 and concluded in July 2025, with approximately 129 participants.3 Participants in the trial will be randomly assigned 1:1 to receive intrathecal apazunersen via lumbar puncture or a sham comparator for 48 weeks.

Original title: “Assessing Antisense Oligonucleotide Apazunersen in Pediatric Angelman Syndrome: The Aspire Study | NeurologyLive - Clinical Neurology News and Neurology Expert Insights”

Yuviwel, a once-weekly injection, will now compete for market share with BioMarin’s lucrative daily shot Voxzogo.

Original title: “Ascendis wins FDA approval of dwarfism drug”

Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one analyst called a "worst case scenario" for UniQure.

Original title: “UniQure says FDA wants another study of Huntington’s gene therapy”

Liver-related side effects have led some analysts to question fenebrutinib’s prospects, even though the drug has now cleared Phase 3 studies in multiple forms of the disease.

Original title: “Roche pill succeeds in another MS study, but approval questions linger”

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.

Original title: “UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism”

Aardvark Therapeutics is down 54% since Friday after the biotech said it detected “reversible cardiac observations” in a healthy volunteer study of its drug to treat extreme hunger in patients with the rare genetic disease.

Original title: “Aardvark Tanks After Pausing Prader-Willi Study on ‘Surprise’ Safety Signals”

Early data show faster viral clearance and favourable safety in mild to moderate COVID‑19

Original title: “Bioxytran reports positive phase 1b/2a results for antiviral ProLectin‑M”

New authorisation extends treatment to children with ISS, SGA and Noonan syndrome

Original title: “FDA approves once‑weekly Sogroya for wider paediatric use”

Yuviwel will compete with BioMarin’s Voxzogo. Meanwhile, BridgeBio is working to bring its own achondroplasia drug, the FGFR3 blocker infigratinib, to the market.

Original title: “Ascendis Enters Achondroplasia Arena as FDA Approves First Weekly Drug”

The detection of certain cardiac issues in a study of healthy volunteers led Aardvark to pause a Phase 3 trial only months before an expected data readout.

Original title: “Safety concerns spur Aardvark to halt key Prader-Willi drug trial”

March 2 (Reuters) - Intellia ... the ‌clinical hold on a late-stage trial of its gene therapy for a heart disease. The gene-editing therapy, nexiguran ziclumeran (nex-z), is being tested separately for a heart ​condition and nerve damage caused by transthyretin amyloidosis, a rare ​and progressive disease resulting from the buildup of a faulty ⁠protein in organs. Sign up here. Last year, the FDA paused Intellia's two trials of the ​gene-editing treatment after a patient ... March 2 (Reuters) - Intellia Therapeutics (NTLA.O), opens new tab said on ​Monday the U.S. Food and Drug Administration has lifted the ‌clinical hold on a late-stage trial of its gene therapy for a heart disease. The gene-editing therapy, nexiguran ziclumeran (nex-z), is being tested separately for a heart ​condition and nerve damage caused by transthyretin amyloidosis, a rare ​and progressive disease resulting from the buildup of a faulty ⁠protein in organs. Sign up here. Last year, the FDA paused Intellia's two trials of the ​gene-editing treatment after a patient in the heart disease study was ​hospitalized with severe liver complications and later died. Intellia Therapeutics said on ​Monday the U.S. Food and Drug Administration has lifted the ‌clinical hold on a late-stage trial of its gene therapy for a heart disease. Evercore analyst Jon Miller said Intellia came off the hold relatively quickly by historical standards, suggesting the overall clinical delay may be limited to roughly three to four months, and called it ​a "great result". The company ​said it ⁠has taken mitigation measures for both trials, such as enhanced liver test monitoring, short-term steroid use for ​early liver issues post-dosing and excluding patients with certain ​liver abnormalities. For ⁠the heart disease trial, Intellia said it is adding new exclusion criteria for patients with recent cardiovascular instability or severely reduced ability to ⁠pump blood.

Original title: “Intellia gets FDA nod to resume second gene therapy trial after safety pause | Reuters”

On the FDA’s docket this month are two decisions pushed back from 2025, including one for a rare form of obesity and another for dry eye disease.

Original title: “FDA Action Alert: BMS, GSK, Aldeyra and More”

The first patient has been dosed in a phase 1/2 trial of GB221, an SMA gene therapy delivered via intra-cisterna magna injection. The US Food and Drug Administration (FDA) has granted the therapy Rare Pediatric Disease designation. FDA approval of GB221 could qualify Gemma Biotherapeutics for a Priority Review Voucher. SMA is a rare neurodegenerative disorder involving progressive muscle weakness and loss of movement. Until 2016, management focused on supportive care and overall quality of life. The identification of SMN1 abnormalities as the underlying cause of SMA enabled development of targeted treatments, 4 of which have obtained FDA approval. The gene therapy utilizes a proprietary serotype hu68 adeno-associated virus vector. It carries a codon-optimized SMN1 transgene, which is contained within an expression cassette modified to limit overexpression and sensory neurotoxicity. CHARISMA is the first clinical trial to evaluate an SMA type 1 gene therapy delivered via the cisterna magna route. GB221 is the first SMA type 1 gene therapy delivered via intra-cisterna magna injection to reach the clinical trial stage. Close more info about First Patient Dosed in Clinical Trial of GB221, Next-Generation Gene Therapy for SMA Type 1

Original title: “First Patient Dosed in Clinical Trial of GB221, Next-Generation Gene Therapy for SMA Type 1”

Aardvark Therapeutics is voluntarily pausing its late-stage trial testing its experimental treatment for a type of rare genetic disease after it found heart-related side effects in healthy volunteers, the company said on Friday. Feb 27 (Reuters) - Aardvark Therapeutics (AARD.O), opens new tab is voluntarily pausing its late-stage trial testing its experimental treatment for a type of rare genetic disease after it found heart-related side effects in healthy volunteers, the company said on Friday. Aardvark was testing ARD-101 as a potential treatment for hyperphagia, or feelings of intense and persistent hunger, in patients with a rare genetic disorder called Prader-Willi syndrome. The company said it is conducting a comprehensive review of the data to inform next steps. "We are committed to advancing the ARD-101 clinical program and we are evaluating optimal therapeutic dosing levels to support its progress," CEO Tien Lee said in a statement. Currently, only one FDA-approved treatment exists for hyperphagia, a once-daily oral pill called Vykat XR by Soleno Therapeutics (SLNO.O), opens new tab. Last year, Acadia Pharmaceuticals (ACAD.O), opens new tab said it will discontinue development of its intranasal therapy to treat Prader-Willi syndrome after it failed to show a benefit in patients in a late-stage trial.

Original title: “Aardvark pauses late-stage trial testing rare disease treatment | Reuters”

Hernexeos is the second drug to secure an FDA approval under the agency’s priority voucher scheme, following in the footsteps of USAntibiotics’ Augmentin XR, which was granted the ticket in December 2025.

Original title: “Boehringer Wins Speedy Lung Cancer Approval Under Commissioner’s Priority Program”