The cost of trials used to support the initial FDA approval of risdiplam (Evrysdi)
Orphan Drugs
Proposal for Addition Of Risdiplam to WHO Model List Of Essential Medicines, for the treatment of Spinal Muscular Atrophy (SMA).
On November 1, 2024, Knowledge Ecology International (KEI) submitted a proposal for the addition of risdiplam to the World Health Organization’s (WHO) Model List of Essential Medicine (EML). You can access the full proposal here: risdiplam2WHOEML1Nov2024 The EML, updated every… Continue Reading
Request for Amendment to add Risdiplam for Spinal Muscular Atrophy (SMA) to Schedule 1 of the Canadian Patent Act
On March 1st, Knowledge Ecology International sent a letter to The Hon. Mark Holland, Ministry of Health, and The Hon. François-Philippe Champagne, Minister of Innovation, Science, and Industry, urging the prompt consideration of adding risdiplam, a drug to treat spinal… Continue Reading
WHA74: KEI’s statement on expanding access to effective treatments for cancer and rare and orphan diseases including cell and gene-based therapies
At the World Health Organization’s 74th World Health Assembly, Knowledge Ecology International delivered the following remarks on expanding access to effective treatments for cancer and rare and orphan diseases including cell and gene-based therapies on Thursday, 27 May 2021. Non-state… Continue Reading
WHO EB 148: KEI’s statement on expanding access to effective treatments for cancer and rare and orphan diseases including cell and gene-based therapies
At the meeting of the World Health Organization’s 148th Executive Board, Knowledge Ecology International delivered the following remarks on expanding access to effective treatments for cancer and rare and orphan diseases including cell and gene-based therapies on Friday, 22 January… Continue Reading
EB 148: WHO board paper on expanding access to effective treatments of cancer and rare and orphan diseases including cell and gene-based therapies
On 16 December 2020 the World Health Organization (WHO) published a paper that delighted in the name, Expanding access to effective treatments for cancer and rare and orphan diseases, including medicines, vaccines, medical devices, diagnostics, assistive products, cell- and gene-based… Continue Reading
KEI Briefing Note 2020:4 Selected Government Definitions of Orphan or Rare Diseases
Zolgensma
Zolgensma is a gene therapy for spinal muscular atrophy (SMA) that was approved by the US FDA in May 2019, at a price of $2.1 million per patient. Zolgensma was licensed from a children’s hospital where it was developed on… Continue Reading
Charity and NIH funding related to Zolgensma
June 14, 2019 Zolgensma, the Novartis brand name for the new $2.1 million gene therapy for treatment of Spinal Muscular Atrophy (SMA), appears to be a remarkable medical breakthrough treatment. It is also, like all of the new cell- and… Continue Reading
Litigation: KEI lawsuit over NIH/Gilead CAR T License Dismissed over Lack of Standing
In an order dated April 11, 2019, Judge Peter J. Messitte granted the NIH’s motion to dismiss for lack of jurisdiction in the lawsuit filed by KEI, which appealed an NIH decision to grant an exclusive license on a CAR… Continue Reading