Blog
RSS FeedPosted by Michael Wonder on 31 Aug 2025
FDA approval of Leqembi subcutaneous formulation charts path to combination therapies for Alzheimer's disease
29 August 2025 - Subcutaneous delivery increases accessibility and introduces potential for at home administration, following model of diabetes and ...
Posted by Michael Wonder on 21 Feb 2025
Harmonising HTA evidence needs and expectations: challenges and opportunities to improve evidence generation, ensure access and affordability
19 February 2025 - Conducting high quality health technology assessments requires high quality evidence. With evolving regulatory standards for faster approval ...
Posted by Michael Wonder on 21 Feb 2025
Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology
19 February 2025 - Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support ...
Posted by Michael Wonder on 21 Feb 2025
FDA approves first treatment for cerebrotendinous xanthomatosis, a rare lipid storage disease
21 February 2025 - Today, the US FDA approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis in adults. ...
Posted by Michael Wonder on 21 Feb 2025
Epkinly (epcoritamab) approved by Japan Ministry of Health, Labour and Welfare for additional indication as a treatment for relapsed or refractory follicular lymphoma
20 February 2025 - Approval based on results from two Phase 1/2 EPCORE clinical trials, which demonstrated strong and durable efficacy ...
Posted by Michael Wonder on 21 Feb 2025
Eylea HD pre-filled syringe with OcuClick dosing system granted marketing authorisation in Canada
20 February 2025 - Bayer is pleased to announce that Health Canada has granted marketing authorisation for Eylea HD (aflibercept injection ...
Posted by Michael Wonder on 21 Feb 2025
Gilead’s seladelpar granted conditional European marketing authorisation for the treatment of primary biliary cholangitis
20 February 2025 - Now approved, seladelpar can provide an important treatment option for people living with the rare liver Disease ...
Posted by Michael Wonder on 21 Feb 2025
EMA validates indication extension application for Aspaveli for treatment of C3G and primary IC-MPGN
20 February 2025 - Sobi and Apellis Pharmaceuticals today announced the EMA has validated an indication extension application for Aspaveli (pegcetacoplan) ...
Posted by Michael Wonder on 20 Feb 2025
PolarityBio receives US FDA breakthrough therapy designation for treatment of Wagner grade I diabetic foot ulcers
20 February 2025 - The FDA has granted breakthrough therapy designation for SkinTE based on key positive data from the Phase ...
Posted by Michael Wonder on 20 Feb 2025
BioRestorative receives FDA fast track designation for BRTX-100 chronic lumbar disc disease program
20 February 2025 - Reflects positive preliminary Phase 2 safety and efficacy data reported to date. ...
Posted by Michael Wonder on 20 Feb 2025
World’s first potential target therapy for diffuse gastric cancer granted fast track designation by the US FDA
20 February 2025 - Signet Therapeutics announced that the US FDA has granted fast track designation for SIGX1094, the world's first ...
Posted by Michael Wonder on 20 Feb 2025
AskBio receives FDA regenerative medicine advanced therapy designation for Parkinson’s disease investigational gene therapy
19 February 2025 - Regenerative medicine advanced therapy designation follows Phase Ib 36 month data. ...
Posted by Michael Wonder on 20 Feb 2025
Harmony Biosciences provides update on the status of the supplemental new drug application for pitolisant in idiopathic hypersomnia
19 February 2025 - Harmony Biosciences today announced that it received a refusal to file letter from the US FDA for ...
Posted by Michael Wonder on 20 Feb 2025
Korea to implement new drug pricing policy that rewards innovation
20 February 2025 - The Ministry of Health and Welfare on Thursday announced a significant policy change in pharmaceutical pricing ...
Posted by Michael Wonder on 20 Feb 2025
Doron Therapeutics receives FDA regenerative medicine advanced therapy designation for Motys for treatment of symptoms associated with osteoarthritis
19 February 2025 - Doron Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 20 Feb 2025
PTC Therapeutics announces FDA acceptance and priority review for vatiquinone NDA for the treatment of children and adults with Friedreich's ataxia
19 February 2025 - PDUFA target action date of 19 August 2025. ...