A conditional approval has meant that Kyowa Kirin and Ultragenyx’ Crysvita can be used in the EU to treat children with a rare, chronic progressive musculoskeletal disorder known as X–Linked Hypophosphataemia (XLH). This disorder forms a skeletal disease that leads to lower extremity deformity and diminished height, where conventional therapy is to counteract excess effects with multiple daily doses of phosphate and active vitamin D. Crysvita is however an anti-FGF23 fully human monoclonal antibody which targets the underlying pathophysiology of the disease. The antibody can now be used in children of just one year old, up to adolescents with growing skeletons. If you would like to read more, please click here for a PharmaTimes article.
Under an accelerated review pathway, Shionogi’s Xofluza has been approved in Japan for use against influenza. This approval makes Xofluza a key contender with the overriding competitor in the antiviral market, Tamiflu, where just one Xofluza pill could replace the 10 required over 10 days for Tamiflu treatment. Another point is that the antiviral resistance developed due to not completing a course of therapy will not be possible as it is just one pill. Roche, who are in a development and sales deal with Shionogi, now plan to follow up that success by rolling it out to the rest of the world as quickly as possible. Please click here if you would like to read further into this with an article from EndPoints.
Sangamo have landed themselves a $3 billion deal with Gilead’s Kite to develop a next-generation of engineered cell therapies for cancer treatments. The deal confirmed is that Kite will use Sangamo’s Zinc-Finger nucleases (ZFN) technology gene therapies to develop autologous cell therapies and allogeneic treatments for certain different cancers. For this, Sangamo will receive $150 million upfront and may receive up to $3.01 billion in potential payments which are related to use of the technology as well as tiered royalties of potential future products. If you would like to read more on this topic, please click here for a PharmaTimes article.