Curated news and analysis on clinical trials, drug approvals, and medical research.
Two Louisiana parents published a children’s book to raise awareness of Infantile Neuroaxonal Dystrophy, a rare disorder affecting two of their daughters. They are also helping raise money for a gene therapy trial.
The FDA issued a Complete Response Letter for Disc Medicine’s bitopertin NDA in EPP, saying additional evidence is needed. The agency wants Phase 3 APOLLO data before deciding.
The FDA granted orphan drug designation to AstraZeneca’s surovatamig for follicular lymphoma. The status supports development for rare diseases but is only an early regulatory step.
The CONDOR randomized clinical trial found brolucizumab better preserved visual acuity than panretinal photocoagulation in proliferative diabetic retinopathy at week 54. Brolucizumab also reduced vision-threatening complications and center-involved diabetic macular edema events, while showing more intraocular inflammation.
Russian lawmakers submitted a bill allowing use of patented drugs in research before patent expiry to prepare generic registration. The measure mirrors a Bolar exemption as patent disputes over early generic launches rise.
A phase I trial and a preclinical study found viral immunotherapy triggered T cell responses in glioblastoma. The clinical study enrolled 41 patients; the mouse study showed prolonged survival and immunologic memory.
A study from the University of Osaka identified the integrin αV–YAP–CTGF pathway linking chronic liver congestion to fibrosis. The findings point to a potential therapeutic target in congestion-related liver disease.
The FDA granted Orphan Drug Designation to Cullinan Therapeutics’ CLN-049 for relapsed/refractory AML. CLN-049 is in Phase 1 studies and had already received Fast Track designation.
The pharmaceutical packaging equipment market is projected to grow from US$6.1 billion in 2024 to US$12.6 billion by 2034. The pharma blister packaging machines market is valued at USD 3.04 billion in 2025 and is forecast to reach USD 4.13 billion by 2035.
OKYO Pharma said it expanded its Scientific Advisory Board with the appointment of Marta Sacchetti as urcosimod advances in neuropathic corneal pain. The company said urcosimod has Fast Track designation and earlier received a single-patient expanded access IND.
The FDA has accepted the New Drug Application for giredestrant plus everolimus in ESR1-mutated, ER-positive, HER2-negative advanced breast cancer. The filing is based on Phase III evERA data showing reduced risk of disease progression or death versus standard-of-care endocrine therapy plus everolimus.
The FDA granted Fast Track designation to COYA 302 for ALS. Phase 1 data showed tolerability and biomarker effects, and the phase 2 ALSTARS trial is underway.
Sensorion appointed Fred Chereau chief executive effective June 1, 2026, while reporting six-month Audiogene trial data for SENS-501. The company said early hearing improvements in cohort 2 persisted at six months and no serious adverse events were reported.
An interim phase 3 analysis showed mezigdomide plus carfilzomib and dexamethasone reduced the risk of progression or death in relapsed/refractory multiple myeloma. The SUCCESSOR-2 study remains ongoing and will assess overall survival and safety.
MSD said sacituzumab tirumotecan improved overall survival and progression-free survival in the Phase III TroFuse-005 trial in endometrial cancer. The study also met its objective response rate endpoint.
The FDA pulled back publication of several studies on COVID-19 and shingles vaccines. The blocked studies involved millions of patient records, and two COVID-19 papers were withdrawn in October 2025.
Regeneron and Parabilis Medicines entered a research collaboration focused on Antibody-Helicon Conjugates. The deal includes $125 million upfront and financing support and up to $2.2 billion in milestones.
The FDA approved Bristol Myers Squibb’s Breyanzi for adults with relapsed or refractory marginal zone lymphoma after at least two prior systemic therapies. In TRANSCEND FL, the CAR T therapy showed a 95.5% overall response rate and 62.1% complete response rate.
Novartis reported Phase III PSMAddition data showing Pluvicto plus standard of care cut the risk of PSA progression by 58% in PSMA-positive metastatic hormone-sensitive prostate cancer. Regulatory decisions in the United States, China and Japan are expected in the second half of 2026.
Drug repurposing expands the therapeutic applications of existing medications, while repositioning examines drugs that never reached market. In neurodegeneration, the approach is described as a pragmatic shortcut into mid- to late-stage trials.
