In this section, is reported the full ATMP news database, sorted by area of interest

One-time dosing with potential lifetime benefit creates challenges for payers to adequately reward the manufacturers of such innovations with a sufficient return on their investment.It is important to characterize the challenges for traditional approaches to assessing the value of one-time gene replacement therapies for the creation of a health economic rationale for a higher value-based cost-effectiveness threshold. The Institute for Clinical and Economic Review in the United States has discussed a range of up to $500K per quality-adjusted life-year gained for ultrarare diseases, and the National Institute for Health and Care Excellence in the United Kingdom has described a variable threshold up to £300,000 per QALY depending on the magnitude of the health gains

Date: Jun 28, 2019

After the obtaining of Marketing Authorisation by EMA in the early days of June, Bluebird Bio annouces a price for Zynteglo, its gene therapy for the treatment of  transfusion-dependent β-thalassemia in patients without donors for hematopoietic stem cell transplantation, of €1.575 millions; payers only pay if Zynteglo works, with a €315.000 advance payment and 5 annual instalments. According to the company,  ability to avoid transfusions and to improve patient’s quality of life justifies the price

Date: Jun 14, 2019

FDA has approved Zolgensma, Novartis gene therapy against spinal muscular atrophy type 1, with a price of $2,125 million per patient. The treatment is the most expensive in history but, according to the swiss Company, the lifesaving benefit justifies the price, although a change in payment and reimbursement methods, in a system without lump sum payment capabilities, wil be needed. FDA uses a clinical trial data for its decision, that shows all of 15 infants treated with the drug were alive and without respiratory assistance at two years old

Date: May 27, 2019

Zolgensma, Novartis one-time gene therapy for the treatment of spinal muscular atrophy, is waiting the FDA market authorization within the month. The swiss company’s CEO said that the price for the gene therapy will be far lower than the rumors of $4-5 millions. The current treatment for SMA is Spinraza, a Biogen product, with a list price of $750000 for the initial year, and $375000 annually thereafter

Date: May 22, 2019
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