The US Food and Drug Administration has approved a new small molecule drug for a rare genetic disease that can impair the supply of thyroid hormone to the brain whilst also leaving excessive levels of the hormone in the bloodstream. The drug, Emcitate (tiratricol), was developed by Egetis Therapeutics US Inc, and received multiple designations in the run-up to its approval on 28 September, including a Priority Review designation in March.
Sofinnova Partners SAS has created a new fund to build and support companies developing medical technologies with plans to launch six to eight new companies over the next five years. This is being enabled by an over-subscribed €82 million financing round announced on 28 September. While the target technologies haven’t been identified, the broad approach is to create companies from the ground up. Explaining this strategy, Antoine Papiernik, managing partner, said the investment group builds and supports “every company from day one.” These assets are then scaled up.
The US Food and Drug Administration has approved a new gene therapy for a rare neurological disorder that manifests at an early age – Sanfilippo syndrome type A. Until the approval, treatment was limited to managing symptoms. The therapy, Fayuvi (rebisufligene etisparvovec), is an adeno-associated serotype 9 virus therapy (AAV9) that delivers a working copy of a gene enabling the body to produce sulfamidase, an enzyme which is missing in patients with the disease. Children with deficient supplies of the enzyme cannot beak down a sugar chain known as heparan sulfate.
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