Aptevo Therapeutics Inc.’s chief scientific officer, Jane Gross, told BioWorld that the sale of the firm’s marketed recombinant hemophilia B therapeutic, Ixinity, to Medexus Pharmaceuticals Inc. allowed for a “cleaner message” to Wall Street. “It was a little difficult to explain having a commercial asset and an R&D pipeline,” she said.
SAN DIEGO – At Biocom's 10th Annual Global Life Sciences Partnering Conference, a panel of players intimately familiar with last year's approval of myelofibrosis treatment Inrebic (fedratinib) explained the backstory of how they got the JAK2 kinase inhibitor off an FDA clinical hold, wrangled the rights to the drug back from the big pharma owner that had acquired the drug from Targegen Inc. and eventually helped the drug gain FDA approval after selling the rights to another large company.
Valrox (valoctocogene roxaparvovec) from San Rafael Calif-based Biomarin Pharmaceutical Inc. moved one step closer to entering the U.S. market, with the company reporting that that the FDA had accepted for priority review the BLA for its investigational AAV5 gene therapy for adults with hemophilia A.
“Lack of knowledge is the true bottleneck to clinical translation. We need to stop telling basic scientists, especially trainees, that their work’s value lies in its translatability.” That is the unexpected advice of none other than William Kaelin Jr., whose scientific discoveries have proved to be both top-rate science and very translatable indeed. His work, for which Kaelin has won the 2019 Nobel Prize in Physiology or Medicine and a host of other awards, has enabled the development of multiple therapies targeting anemia and cancer, including vadadustat.
Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages. It is not surprising that, according to the American Society of Hematology, the FDA approved several new therapies – or new indications for previously approved therapies – in 2019 for people living with non-malignant blood disorders. Those included two disease-modifying treatments for sickle cell disease and the first anticoagulant for venous thromboembolism management in children.
No matter how effective it is, a drug is worthless if the people who need it can’t afford it. That’s been almost an anthem for patients and policy wonks testifying before U.S. Congress on drug prices.
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