New Products
Big pharma is under pressure to find the next big thing, and new launches bring high hopes. But focusing on the number of new drugs approved each year may mislead on the sector’s success if profitability and significant sales elude a majority.
Throughout 2024, Chinese firms as sole sponsors initiated a total of seven global Phase III trials that including US sites. BeiGene, Genfleet and InnoCare are expected to add four similar studies this year.
Just a handful of big pharma approvals of 2024 are tipped to be multi-billion dollar blockbusters. Even though it is still early days for these drugs, the pull of M&A is looking increasingly strong for big pharma as more smaller firms go it alone.
The year’s new drugs included the first drug approved for non-alcoholic steatohepatitis (NASH) and two drugs for primary biliary cholangitis (PBC).
Vertex hopes to convert CF patients to new combo Alyftrek from Trikafta with once-daily dosing. Approved on 20 December, the new triplet has longer patent protection and 7% higher US pricing.
The antisense oligonucleotide specialist is ready to launch its rare disease therapy Tryngzola but could face competition from a more potent rival by mid-2025.
The cell therapy won approval for pediatric steroid-refractory acute graft versus host disease after two complete response letters over the last four years.
Nemluvio is the first IL-31 inhibitor approved by the US FDA for atopic dermatitis and will compete with Sanofi/Regeneron’s Dupixent and other, newer IL-13 inhibitors.
China’s fresh approval of a sublingual form of Simcere's edaravone/dexborneol brain cytoprotective agent for acute ischemic stroke is paving the way for an international Phase III study in this indication.
The US FDA approved the TTR-stabilizing drug for ATTR-CM, with the drugmaker pricing it below that of Pfizer’s Vyndaqel/Vyndamax.
The agency granted accelerated approval to zanidatamab in HER2-positive biliary tract cancer, though the company is eyeing larger indications for the drug.
Astellas's C5 inhibitor faces another approval challenge, this time for a supplemental filing in the US, following the withdrawal of a European submission, although it continues to do well in what is its only market so far.
The drug maker anticipates a potential label expansion for Revuforj (revumenib) from KMTA2-translocation acute leukemias into NPM1-mutant AML.
Following ex-US approvals, PTC obtains a US OK for Kebilidi, branded Upstaza outside the US, as a gene therapy for ultra-rare AADC deficiency. The drug is administered via neurological surgery.
Sales of its novel COPD therapy in its first quarter on the market were more than double consensus estimates, and Verona sees more to come.
Doubling some analyst projections for its second full quarter on market, Madrigal’s NASH drug appears headed to blockbuster status and a sustained market position.
The US FDA approved Orlynvah for uncomplicated urinary tract infections among adult women, despite some concerns about the potential for off-label use and resistance.
Pfizer beat GSK with a new indication for its RSV vaccine in high-risk individuals aged 18-59.
An exec said the company is confident it can ensure testing for the biomarker becomes routine for the drug, which is the first FDA-approved medicine to target CLDN18.2.
Vyalev, a 24-hour constant infusion of carbidopa and levodopa prodrugs, obtained US FDA approval and will launch at $119,000 per year.