Becoming the first type II RAF inhibitor for relapsed or refractory BRAF-altered pediatric low-grade glioma, Day One Biopharmaceuticals Inc.’s Ojemda (tovorafenib, DAY-101) gained U.S. FDA accelerated approval on April 23, a week earlier than its expected PDUFA date, bringing the Brisbane, Calif.-based company a rare pediatric disease priority review voucher.
An oral Bruton’s tyrosine kinase (BTK) inhibitor that Sanofi SA acquired in 2020 through its $3.68 billion buyout of Principia Biopharma Inc. is headed toward regulatory filings in the U.S and EU by the end of the year, following phase III data in immune thrombocytopenia.
Skyhawk Therapeutics Inc. signed on to work with Ipsen SA in a neurological disease-focused deal worth potentially $1.8 billion to discover and develop novel small molecules that modulate RNA. The deal is an option agreement for exclusive global rights to two candidates. Once the parties have identified and validated those candidates, Paris-based Ipsen will take on all further development and commercialization.
The Pharmaceutical Research and Manufacturers of America (PhRMA) is not happy with the Biden administration or the new 340B rule, finalized on April 18 by the U.S. Department of Health and Human Services (HHS), concerning the dispute resolution process put in place in 2020.
Robust top-line phase III data showed Intra-Cellular Therapies Inc.’s marketed oral atypical antipsychotic drug, Caplyta (lumateperone), achieved statistically significant and clinically meaningful best-in-class results, potentially expanding its reach into the billion-dollar major depressive disorder market. The company’s shares (NASDAQ:ITCI) surged to a 52-week high of $84.89 throughout the day, closing at $79.84, up 23.3%, or $15.08, on April 16.
The $27.9 billion raised through biopharma follow-on offerings in the early months of 2024 has hit industry records never seen before. So far in three-and-a-half months in 2024, the industry has raised more than was raised during 21 of the past 24 full years.
Following decisions in 2023 to back away from its lead neurological therapies and conserve cash, Eliem Therapeutics Inc. found a vote of investor confidence and a new direction through its acquisition of privately held Tenet Medicines Inc. and its anti-CD19 antibody for autoimmune disease.
With two respiratory syncytial virus (RSV) vaccines approved by the U.S. FDA in 2023 and a third nearing its May PDUFA date, decades of research has finally provided infants and older adults protection from the disruptive and sometimes deadly virus. But what about people in the middle, particularly those with certain chronic medical conditions? New York-based Pfizer Inc. rolled out phase III data April 9 showing that its approved RSV vaccine, Abrysvo (RSVpreF), met primary endpoints in adults ages 18 to 59 who were at high risk of RSV.
In a long and winding regulatory road that began with two complete response letters 15 years ago, Allschwil, Switzerland-based Basilea Pharmaceutica Ltd. finally gained U.S. FDA approval of its intravenous cephalosporin antibiotic, Zevtera (ceftobiprole medocaril sodium), to treat Staphylococcus aureus bacteremia, acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia.
Jumping in for the first time to the hot antibody-drug conjugate (ADC) space, Paris-based Ipsen SA pulled in exclusive global rights to a preclinical ROR1-targeting candidate from Sutro Biopharma Inc. in a deal worth up to $900 million. STRO-003, the first ADC to join Ipsen’s portfolio, contains an anti-ROR1 human IgG1 antibody (SP-11385) conjugated to an exatecan warhead, or payload.
