Eli Lilly has delayed the submission of an application for approval of donanemab, an Alzheimer's treatment, from the end of March to the end of the year. This decision comes after a draft decision by Medicare to limit coverage of Alzheimer's treatments. Biogen's Aduhelm, which has the same treatment mechanism as donanemab, did not show enough therapeutic effect in clinical trials. As doubts over the drug's effectiveness grow, Medicare’s draft policy will limit coverage for both treatments to those enrolled in randomized, placebo-controlled clinical trials, if finalized later this year.
Eli Lily가 알츠하이머 치료제 donanemab의 승인 신청서 제출 일정을 기존 3월 말에서 연말로 연기했습니다. 이 결정은 메디케어의 알츠하이머 치료제에 대한 보장을 제한하는 정책 초안 내용을 반영해 내려졌습니다. donanemab과 동일한 치료 기전을 가진 Biogen의 Adulhelm이 임상 시험에서 치료 효과를 보이지 못하고, 약물 효과에 대한 의심이 커지면서, 메디케어는 두 치료제에 대한 보장을 randomized, placebo-controlled 임상 시험에 등록한 사람들로만 제한한다는 내용의 정책 초안을 발표했습니다. Original Article: BioPharma Dive.Lilly slows FDA submission of Alzheimer's drug after Medicare decision
23andMe announced that 23andMe and GlaxoSmithKline (GSK) decided to extend their drug target discovery collaboration for another year. Since starting their partnership in 2018, the two companies have identified more than 40 therapeutic programs and have advanced a CD96-targeted immuno-oncology drug to the clinical development stage. GSK will be responsible for the later clinical phases of the CD96 drug, and 23andMe will receive royalties in a Phase 1 program.
A study on the therapeutic effect of lisdexamfetamine, a stimulant used in the treatment of ADHD, for KCNMA1-linked channelopathy was published. KCNMA1-linked channelopathy is a rare disease that causes movement disorders and has so far been identified in 75 patients worldwide. This paper was published in the ‘Movement Disorders Clinical Practice Journal’.
The story of patients who participated in a clinical trial to treat rare diseases using CRISPR, a gene-editing technology, was introduced in The Washington Post. CRISPR-based therapies are being tried to treat ATTR amyloidosis and Duchenne muscular dystrophy. Researchers are also working on a CRISPR-based platform to transform red blood cells into factories that produce proteins that are not present in the blood.
Researchers at the University of California have developed a test, called AS-NMD assay, that can measure cellular nonsense-mediated RNA activity (NMD) activity, used to determine the effect of 704 FDA-approved drugs on NMD. Researchers say that 20% of genetic diseases can be treated by targeting NMD, so it is important to study the mechanism of NMD-modulatory drugs.
Since its formation in 2011, the International Rare Disease Research Consortium (IRDiRC), the largest rare disease research consortium in the world which includes nearly 60 organizations, has identified the genetic causes of more than 4,000 rare diseases and has helped develop 200 treatments.
Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…