EC approves BioMarin drug as first treatment for children with common form of dwarfism; Servier sNDA wins approval

27 Aug 2021
endpts.com
AntibodyBiosimilarSmall molecular drugCollaborate
The European Commission just approved its first drug for use in children with achondroplasia, a common form of dwarfism. BioMarin received marketing authorization today for Voxzogo, also known as vosoritide. The drug is a once-daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty. An estimated 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia — and a third of this population are in countries authorized under the EMA license. The French National Agency for Medicines and Health Products Safety also granted Voxzogo a “Temporary Authorization for Use” to allow immediate access. The list price in France under that temporary authorization is about $840 per vial across all ages and weights. Annually, that would be just over $300,000, assuming a patient takes a dose every day. French biotech Servier announced today that they won approval from the FDA for another indication of their oncology drug Tibsovo. The new indication is for the treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation, which is a rare type of cancer in the bile ducts in the liver. Tibsovo has previously been approved to treat AML. The approval is a win for Servier, which spent $1.8 billion late last year to acquire Tibsovo from Agios, along with the rest of their pipeline. Servier came under fire earlier this year after they were fined $3.2 million and convicted on manslaughter, involuntary wounding and aggravated deception charges in a French court. This happened after French prosecutors associated Servier’s diabetes drug Mediator with up to 2,000 deaths in the 33 years the drug was on the market in France. Chinese AI startup Insilico and 4B Technologies announced today that they have agreed to a strategic collaboration. The goal? To develop small molecule therapies for ALS and other neurological diseases. According to 4B Technologies CEO Xiaoming Guan, the traditional R&D process is time-consuming and expensive. As such, collaborating with a company that has an emphasis on AI would allow 4B to expedite its R&D process. This is not Insilico’s only recent deal. Just a few weeks ago, they announced a deal with BioNova to jointly develop small molecule inhibitors. This is just two months after Insilico CEO Alex Zhavoronkov pulled in a massive $255 million Series C round back in June. Innovent Biologics and Bolt Biotherapeutics announced that they are collaborating on three new immune-stimulating antibody conjugate candidates. The oncology targets remain undisclosed. The deal will see Innovent taking responsibility for all R&D costs through clinical proof-of-concept — and utilizing Bolt’s ISAC platform to help create the candidates. Upon review, the companies can exercise licensing options for continuing development and exclusive commercialization rights. According to the agreement, Innovent has the rights to all three candidates in China, Hong Kong, Macau and Taiwan. Bolt will receive $5 million in cash upfront from Innovent and a possible future equity investment of up to $10 million. Both companies have additional milestone payments and royalties based on development and commercialization in different markets. Bolt, based out of California, also partnered with Genmab in June to develop up to three new antibody-based conjugates in oncology. Chinese biopharma firm Bio-Thera Solutions and British pharmaceutical company Hikma Pharmaceuticals have entered into a license agreement to commercialize a biosimilar to Janssen’s Stelara, an immunosuppressant approved in the US for Crohn’s disease. The biosimilar candidate is currently in a global Phase III clinical trial and under the agreement, Bio-Thera will keep responsibility for the development, manufacturing, and supply of the potential biosimilar. Hikma will have exclusive rights to commercialize it in the US, and first-right-to-negotiate to add Europe, excluding the nine members of the Commonwealth of Independent States. Bio-Thera is eligible for an upfront payment of $20 million, as well as further development and commercial milestones of up to $130 million.
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