Diseases

BPOM is drafting a conditional approval route for the M72 TB vaccine and a joint review with the European Medicines Agency, ahead of Phase 3 results.

INFARMED recommends public funding for Refixia nonacog beta pegol in children under 12 with haemophilia B, based on two single-arm trials. #SyenzaNews #MarketAccess

SMA Europe has relayed Novartis's figures on tumour reports in patients previously treated with Zolgensma, including two cases of vector DNA integration.

The G-BA has found no proven added benefit for dupilumab in chronic spontaneous urticaria, compared with omalizumab plus second-generation H1 antihistamines.

"Truqap prostate cancer withdrawal: AstraZeneca pulled its EU bid for newly diagnosed use after EMA raised benefit-risk concerns." #SyenzaNews #MarketAccess

A Mayo Clinic cardiologist's TV-set question makes the case for a home blood pressure monitor, with a caution on device reliability and home data in the chart.

The Dutch health institute Zorginstituut Nederland has concluded that a second cochlear implant for adults with severe hearing loss in both ears belongs in the basic health package. #SyenzaNews #MarketAccess

FDA has finalized its potency assurance guidance for cell and gene therapy products. It covers lot-release testing, potency assays, acceptance criteria and how sponsors should build a strategy from the IND stage onward. #SyenzaNews #CellAndGeneTherapy #FDA

Dr. Reddy's has FDA tentative approval for generic lenacapavir HIV prevention, priced at US$40 per person per year across 120 countries. #SyenzaNews #HIVprevention #MarketAccess

EMA's safety committee has opened a Deanxit safety review at Belgium's request, after reports of movement disorders and memory problems with the flupentixol/melitracen combination. The Belgian agency also questioned the benefit of combining the two substances. #SyenzaNews #Pharmacovigilance #EMA #MentalHealth

The FDA has granted accelerated approval to Voceryq, the first plasmid DNA-based immunotherapy in the US, for adults with recurrent respiratory papillomatosis.

Infarmed's first Deep Dive looks at migraine medicines in Portugal. In 2025, more than 73,000 people used SNS-reimbursed migraine therapies, 85% of them women. Patients paid about €6 million (61%) of the €9.9 million pharmacy bill, and atogepant, funded only at the end of 2024, already led spending. #SyenzaNews #Migraine #MarketAccess #Portugal