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- Scotland becomes the first part of the UK to offer routine newborn screening for spinal muscular atrophy (SMA) as part of the standard heel prick test performed around four days after birth. - The two-year evaluation, funded by the Scottish government and Novartis with £530,400 total funding, will assess early detection capabilities for SMA, which affects three to four babies annually in Scotland. - Early pre-symptomatic treatment offers the best chance for children to follow typical developmental pathways, with three disease-modifying therapies now available through NHS Scotland. - Results from this evaluation will inform the UK National Screening Committee's decision on whether to permanently include SMA screening in the national newborn screening programme.
- The Scottish Medicines Consortium has approved Rybrevant (amivantamab) in combination with carboplatin and pemetrexed as the first targeted therapy specifically for first-line treatment of advanced NSCLC with EGFR exon 20 insertion mutations in Scotland. - The Phase 3 PAPILLON study demonstrated that the combination increased median progression-free survival by 4.7 months (11.4 vs 6.7 months) compared to chemotherapy alone, with a 73% objective response rate. - This approval addresses a significant unmet medical need for patients with EGFR exon 20 insertion mutations, who represent 12% of all EGFR mutation-positive NSCLC cases and historically have limited treatment options. - The decision marks an important milestone for approximately 5,300 lung cancer patients diagnosed annually in Scotland, with NSCLC accounting for 80-85% of all cases.
- Ananda Pharma announced plans to delist from AQSE Growth Market and transition to private company status, citing high regulatory costs and limited liquidity that consumed resources better deployed in clinical development. - The company terminated its involvement in a landmark NHS-funded epilepsy trial at UCL and Great Ormond Street Hospital after contractor supply issues, allowing focus on its lead asset MRX1 for pain indications. - Phase 1 results for MRX1 showed favorable safety profile with only mild adverse events across 20 participants, supporting advancement to Phase 2 trials for endometriosis and chemotherapy-induced peripheral neuropathy starting Q1 2026.
- The Scottish Medicines Consortium rejected donanemab (Kisunla) for NHS Scotland use, citing uncertainty around the drug's modest clinical benefit despite UK regulatory approval in October. - This marks the second Alzheimer's drug rejection by Scottish authorities in three months, following a similar decision on lecanemab (Leqembi). - Alzheimer Scotland advocates are calling for a specialized Dementia Drugs Fund and reformed assessment processes, arguing current evaluation methods are inadequate for addressing the UK's leading cause of death. - Donanemab works by targeting amyloid protein buildup in the brain through antibody infusions, potentially slowing Alzheimer's progression by up to two years in some cases.
- Elranatamab and teclistamab, bispecific antibodies, have been approved by NHS Scotland for myeloma patients who have undergone at least three prior treatments. - Clinical trials show these drugs can halt myeloma progression for an average of 11 months and induce complete remission in some patients who previously did not respond to treatment. - These treatments represent the first new class of myeloma drugs approved in Scotland in nearly seven years, offering a new lifeline for patients with limited options. - The approvals are based on data from the MajesTEC-1 and MagnetisMM-3 trials, demonstrating overall response rates of 63% and 61% for teclistamab and elranatamab, respectively.
