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Vertex has top orphan asset in the pipeline, as pharma eyes shiny obesity drugs: report
2024-04-24
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Vertex has only just nabbed the title of top orphan pipeline drug after Merck & Co.'s sotatercept was approved.
Vertex’s cystic fibrosis triple combo is poised to become the top-sellinMerck & Co.ug sotatercept net present value, amid shifting sentiments in the pharma industry for rare disease drugs, according to Evaluate.
Vertex, ocystic fibrosisiple,” which combines a new molecule called vanzacaftor with Vertex’s already approved CF meds tezacaftor and deutivacaftor, now heads up Evaluate’s new list of orphan drugs expeEvaluatebe at the top in four years' time. The med has a net present value of $5.8 billion, with $1.26 billion in sales expected by 2028.
VX-121for the trvanza triplet into Vertex’s own market share with thvanzacaftorrikaftVertexch is listed as the second-mtezacaftorle ordeutivacaftorfor approved meEvaluatesales that could top $6 billion in that year. But Vertex can expect some savings, as the triple has fewer royalty payouts attached, according to Evaluate.
Vertex has only just nabbed the covVertexitle after Merck & Co.’s sotatercept Trikaftaoved, removing it from the pipeline section of Evaluate’s annual ranking. That drug had a net present value of $9 billion and is now kVertexs Winrevair. The drug did not make the approved orphan drug list with a projected $2 billEvaluateales for 2028. However, Merck has a few more indications up its sleeve that could boost those expectations with additional approvals down the line.
Vertex//public.flourish.studio/visualisation/1767542Merck & Co.sotaterceptEvaluateMerck
Behind Vertex is Johnson & Johnson’s nipocalimab, which the company refers to as a “pipeline in a product.” The drug just got an orphan drug tag in fetal and neonatal alloimmune thrombocytopenia (FNAIT), but that’s not the indication that helped land it in the number 2 slot on the pipeline list. Evaluate instead cited its more advanced indication, myasthenia gravis, a market that is growing ever more crowded. Nipocalimab’s net present value is $3.60 billion with predicted 2028 sales of $737 million.
Gene edVertexbiotJohnson & Johnsonapenipocalimabed the third position with a net present value of $3.50 billion and sales of $891 million for 2028, fetal and neonatal alloimmune thrombocytopenia (FNAIT)ed, Intellia would be the first company to have a drug approved that edits the cell’s genome wiEvaluate body.myasthenia gravisNipocalimab
Late last year, CRISPIntellia Therapeuticsrtex earned approval for Casgevy in sickle cell disease. The treatment uses CRISPR-Cas9 to edit stem cells outside the body and then put theRegeneron to express theIntelliaersion of hemoglobin. So it was the first gene editing approval, but a slightly different modality than what Intellia and Regeneron are going for.
Cancer drugs froCRISPR Therapeuticsen taVertex final two slots on tCasgevy.sickle cell diseaseIntelliaRegeneron
Overall, orphan drugs are expected to bring in $185 billion in sales this year, rising to $270 billion by 2028, according to Evaluate. But growth in orphan drug sales is expected to slow into the single digits after averaging 11% growth for the 10 years leading up to 2023. Evaluate blames this deceleration on the “blazing return” of huge drugs in major diseases. Read: obesity.
AObesitymajor interest is neuroscience where major breakthroughs have occurred, and huge deals are being made to capture the biotechs working in the space. Bristol Myers Squibb bought Karuna Therapeutics and its schizophrenia med KarXT for $14 billion while AbbVie picked up Cerevel for $8.7 billion, all at the end of 2023.
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