Rare Diseases
The European Medicines Agency is set to adopt opinions on whether marketing approval should be granted to five new products, including three orphans. A decision is also due on a previously-rejected Alzheimer’s drug that was under re-examination.
The US FDA’s Rare Disease Hub is becoming more concrete, with dedicated 2026 funding and momentum for a more substantial user-fee allocation going forward.
The draft report lays out a unified EU framework to reduce disparities and modernize rare disease diagnosis, treatment, care and research.
The consolidation of HR and other services into one unit may require PDUFA funding system changes, and industry rejects the FDA’s proposal to limit the orphan drug fee waiver.
Pink Sheet editors discuss the direction of the FDA and CBER after Vinay Prasad’s exit, CBER’s similarities to the Harry Potter saga, as well as the Real-Time Oncology Review pilot’s contributions to Commissioner’s National Priority Voucher approval times.
Daiichi Sankyo’s MMR Vaccine, Ultragenux’s triheptanoin also won approval recommendations.
The FDA’s contrasting decisions on leucovorin and idebenone reveal a tightening regulatory stance where only therapies showing strong mechanistic rationale and large, credible treatment effects can overcome the limitations of nontraditional evidence sources.
The agency requested uniQure test AMT-130 against a control arm in which patients undergo a sham procedure. The treatment may be more akin to other neurodegenerative gene therapies than the Huntington's chorea drugs that were approved based on placebo-controlled trials.
Neonatal drug development struggles with the lack of a standard of care and pervasive off‑label use, but the FDA’s growing preparedness for AI and real‑world evidence approaches offers an opening to harness NICU data to accelerate neonatal‑focused therapies.
Pink Sheet editors discuss the generic and biosimilars industries’ 2026 priorities as well as FDA Commissioner Martin Makary’s interesting take on the compassionate use program.
During a Senate hearing, rare disease stakeholders said the FDA is not engaging with patients as much as before and suggested it may be hindering drug development.
The FDA commissioner said he signed all compassionate use requests that came to his desk, even though the career staff traditionally handle them.
Planning for evidence generation to support efficacy and safety ideally should begin as soon as the patient and genetic target are identified, and clinical results should be robust given the very small sample size for individualized treatments, the agency said in the new draft guidance.
Moderna’s mRNA-1010 is an outlier among RTF recipients, which are dominated by rare disease and neuroscience candidates, such as Axsome’s fibromyalgia drug and Neuvivo’s ALS immunotherapy.
Disc Medicine's bitopertin, the first novel agent with an FDA Commissioner's National Priority Voucher to complete a review, failed to show biomarker endpoint was associated with clinical benefit, the complete response letter states.
Despite continually pledging flexibility in rare pediatric drug development, several recent FDA complete response letters suggest a growing insistence on traditional evidence standards that clashes with the realities of ultra‑rare disease research.
The US Labor Department also added a proposed rule requiring PBMs to disclose additional compensation information to certain employer-plan sponsors.
Newly revised regulations for the implementation of China’s Drug Administration Law transition to a market authorization holder/product-centered regulatory approach in response to the rapidly changing biopharma environment.
It is “integral” that a planned increase in genomic testing supports access to treatments such as advanced therapies, says England’s chief scientific officer. Blood cancer and rare diseases are among the conditions expected to benefit from the country’s testing push.
Changing regulatory standards for cell and gene therapies extend the flexibility with manufacturing while the tabelecleucel complete response letter showed high expectations for rigor in clinical trials.