Takeda and Evozyne signed a $400 million contract to develop gene therapy for rare diseases. If Evozyne's AI and machine learning technology discovers a therapeutic sequence, Takeda will own the license to develop and commercialize it as gene therapy.
FDA approved Novartis Vijoice (alplisib) as the first treatment for PIK3CA-Related Overgrowth Spectrum (PROS). PROS is a rare disease that causes atypical overgrowths and anomalies in blood vessels, with a prevalence of about 14 per million people. Conditions associated with PROS include KTS, CLOVES syndrome, ILM, etc.
Infants admitted to the NICU after birth account for 10-15% of all newborns, and most of the cases are due to premature birth, but there are also many cases of genetic diseases. In this article, the background story of the rapid Whole Genome Sequencing-based screening test for infants and the research results of GeneDX's rWGS screening test were introduced.
The Institute for Clinical and Economic Review (ICER), an unofficial US drug price watchdog, has published a white paper examining the benefits and risks of policy reforms related to rare disease drugs. This 47-page publication introduces the positive effects and potential risks of rare disease support policies since the passage of the Orphan Drug Act (ODA) in 1983 on the development of treatments for rare diseases to date. It also suggests various policy reform directions to overcome risks.
비공식 미국 약가 감시 단체인 ICER(Institute for Clinical and Economic Review)가 희소 질환 의약품과 관련된 정책 개편의 이점과 우려사항을 조사한 백서를 발간했습니다. 47페이지에 이르는 이번 발간물에는 1983년 ODA(Orphan Drug Act) 통과 이후 희소 질환 지원 정책들이 현재까지의 희소 질환 치료제 개발에 미친 긍정적 효과와 잠재적 위험에 대한 조사 결과가 담겨 있습니다. 그리고 위험을 극복하기 위한 다양한 정책 개선 방향을 제시하고 있습니다. Original Article: ICER.Evaluating Reforms to Orphan Drug Development, Pricing, and Coverage
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