Over the past decade, three drugs—Biogen’s Spinraza, Roche’s Evrysdi, and Novartis’ gene therapy Zolgensma—have transformed spinal muscular atrophy (SMA) treatment. However, unmet needs remain. Biogen, the first to receive SMA approval in 2016, is addressing this with a higher dose of Spinraza (nusinersen). In Biogen’s phase 2/3 DEVOTE study, the higher dose significantly improved motor…
First patient dosed in Phase 3 study of oral cladribine in gMG
29 August, 2024: Merck has announced the first patient has been dosed in the Phase III MyClad trial (NCT06463587) evaluating the efficacy and safety of oral cladribine for the treatment of generalized Myasthenia Gravis (gMG). Cladribine capsules have the potential to be the first oral treatment for gMG patients. gMG is a rare, neuromuscular disorder…
Tolebrutinib meets primary endpoint in HERCULES phase 3 study
First and only to show reduction in disability accumulation in non-relapsing secondary progressive multiple sclerosis 2 September 2024: Positive results from the HERCULES phase 3 study showed that tolebrutinib, Sanofi’s oral brain-penetrant BTK inhibitor, met the primary endpoint of improvement over placebo in delaying time to onset of confirmed disability progression (CDP) in people with…
Ubrelvy can prevent migraines when used early
AbbVie’s Ubrelvy (ubrogepant) — which is approved to treat migraines – can also help to prevent them when used before the onset of headache pain, according to a recent study from Neurology journal. The phase 3 PRODROME Trial suggests that Ubrelvy was 73% more effective in preventing a migraine attack than a placebo. In the…
Leqembi® (lecanemab) authorised for early Alzheimer’s disease in Great Britain
In Great Britain, lecanemab is indicated for the treatment of mild cognitive impairment and mild dementia due to Alzheimer’s disease (AD) in adult patients that are apolipoprotein E ε4 (ApoE ε4)* heterozygotes or non-carriers1 Great Britain becomes the first country in Europe to authorise the medicine, which targets an underlying cause of AD1 Authorisation based…
CREXONT for Parkinson’s disease – Amneal’s capsules gain FDA approval
August 8th 2024: The US Food and Drug Administration (FDA) has granted approval to Amneal Pharmaceuticals‘ CREXONT (carbidopa/levodopa) extended-release capsules for the treatment of Parkinson’s disease. This novel oral formulation combines immediate-release granules and extended-release pellets, meaning that therapeutic levels of levodopa and carbidopa may be maintained with less frequent dosing to maximise “good on” time,…
Valiltramiprosate -oral Aβ-targeting therapy for Alzheimer’s disease
American clinical-stage biopharmaceutical company Alzheon has recently presented promising interim results from its Phase II Biomarker Trial (NCT04693520) of its oral, brain-penetrant, amyloid-oligomer inhibitor ALZ-801 (valiltramiprosate) at the American Academy of Neurology 2024 annual meeting. ALZ-801 has the potential to be the first oral Aβ-targeting therapy to be approved in the US and EU in…
First-in-class therapy Cebranopadol for treatment of pain
Results shared at International Association for the Study of Pain (IASP) 2024 World Congress on Pain demonstrate that cebranopadol produces potent and prolonged pain relief with 25% less respiratory depression and significantly slower onset of effect with fewer respiratory adverse effects compared to oxycodone Findings highlight therapeutic potential of cebranopadol, an investigational treatment with a…
Alzheimer’s drug lecanemab rejected by EMA
On 25 July 2024 the European Medicines Agency (EMA) rejected a licence for lecanemab to treat Alzheimer’s disease. The EMA said the benefits did not counterbalance the risk of serious side effects, especially bleeding and swelling in the brain. The MHRA in the UK is still considering whether to grant a licence, and a decision is…
