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.
New analysis from Clarivate Analytics' Cortellis Forecast Team predicts 11 medicines set to enter the market in 2020 will reach more than $1 billion in sales by 2024.
Data for this report were compiled from Cortellis, the suite of life sciences intelligence solutions from Clarivate Analytics. Cortellis includes the broadest and deepest range of sources of intelligence across the R&D lifecycle, including annual filings, drug pipelines, clinical trials, patents, chemistry, deals, conferences and company announcements.
Crowned by a potential cure for severe hemophilia A, that could become the most expensive drug ever, a new list of 11 medicines expected to generate $1 billion-plus in annual sales by the end of 2024 or earlier throws into stark relief the growing tension between medical innovation and society's ability to pay for it. The 2020 Cortellis Drugs to Watch list, including medicines both approved and likely to be, points to a future of ongoing conflict between payers and industry spurred by fundamental disagreements.
LONDON – Freeline Therapeutics Ltd. believes it has found the dose at which FLT-180a, its gene therapy for hemophilia B, will provide a functional cure, promoting expression of factor IX (FIX) blood clotting factor within the normal range.
Emendo Biotherapeutics Inc. CEO David Baram told BioWorld his firm’s allele-specific gene-editing approach offers such an advantage over previous methods that “we decided to take the challenge of curing diseases that require the highest precision possible,” and the New York-based firm bears an impressive list of partners. “Doors opened immediately and collaborations formed very fast,” sometimes “even faster than we could digest,” he said.
Ten days after its JAK1 inhibitor, itacitinib, failed a phase III trial in acute graft-vs.-host-disease (GVHD), Incyte Corp. has finalized a deal that could provide an alternative growth path. It is paying Morphosys AG $750 million up front, investing $150 million in its stock and is on the hook for up to $1.1 billion in milestones for a 50% interest in U.S. rights to the CD19-targeting antibody tafasitamab (MOR-208) and for 100% of the rights in all other territories.
Apellis Pharmaceuticals Inc.’s CEO, Cedric Francois, said his firm’s phase III study called Pegasus testing pegcetacoplan, or APL-2, in paroxysmal nocturnal hemoglobinuria (PNH) “finally established that there is an important unmet medical need in this disease.”
Nearly four years after its start, a phase III trial of Gamida Cell Ltd.'s ex vivo expanded cord blood candidate, omidubicel, for hematologic malignancies is fully enrolled, the company said.
DUBLIN – Gene therapy firm Freeline Therapeutics Ltd. secured the first $40 million tranche of an $80 million series C round from its founding investor and principal shareholder Syncona plc to generate further data from its two clinical-stage programs, in hemophilia B and Fabry disease, to fund expansion of its team and to continue the ongoing buildout of its manufacturing operations in Munich.