Project name: pomalidomide
Disease: Multiple myeloma
Peak sales estimate: $1.1 billion
Approved: Feb. 8
The FDA last year green-lighted Pomalyst, or pomalidomide, against cases of multiple myeloma that have persisted after two or more prior therapies. Those prior therapies include market-leading Velcade from Johnson & Johnson ($JNJ) and Japan's Takeda, as well as Celgene's ($CELG) own Revlimid, its best-selling drug, which last year had sales of $3.8 billion.
With approval of Pomalyst came forecasts the oral treatment would hit peak sales of $1 billion or better. It also came less than a month after Celgene CEO Bob Hugin drew industry attention with his plan to double sales of the biotech to $12 billion by 2017. Pomalyst figures into that plan and the approval provided a quick "aha" moment that Hugin's pledge was achievable. With another blockbuster in the making, Pomalyst also helps Celgene move beyond its heavy reliance on Revlimid, which in 2012 accounted for about two-thirds of its revenues.
The FDA based its approval of Pomalyst on a clinical trial of 221 patients in which 7.4% of patients on Pomalyst alone had their multiple myeloma at least partially wiped out. When patients put on a regimen of Pomalyst plus low-dose dexamethasone, 29.2% achieved the objective response rate, with a 7.4-month median duration of response. The FDA considered those results promising enough for Celgene to go to the market with the drug while it did further study to confirm its safety and efficacy.
But Pomalyst was preceded in the market by another drug the FDA approved as a last ditch effort against multiple myeloma. Kyprolis (carfilzomib) from Onyx Pharmaceuticals was approved 7 months earlier. Both drugs won orphan drug designation and both come with significant price tags. Celgene priced Pomalyst at $10,500 monthly, slightly higher than the $9,950 per month charged for Kyprolis.
Both drugs have also been selling well in a market that is slated to only get bigger, powered in part by their approvals. The market has been forecast to grow 60% by 2021, reaching more than $7 billion from about $4.4 billion. But it is also getting very competitive. The success of Kyprolis quickly drew notice and was a key driver for Amgen ($AMGN) in August to offer $10.4 billion to buy Onyx. Takeda has no intentions of meekly surrendering its leading positions in the treatment category, however. Takeda's Millennium has disclosed positive mid-stage data on its successor to Velcade, MLN9708, which has already moved into Phase III. Of the 52 patients treated in Phase II, 58% demonstrated at least a 90% reduction of myeloma proteins in their blood. Remissions was reported in nearly 25% of the patients receiving MLN9708. -- Eric Palmer (email | Twitter)
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