Dive Brief:
- Celgene Corp. has secured approval for a first-of-its-kind cancer medication, aiding the big biotech's pursuit of a more diverse group of marketed products.
- The Food and Drug Administration gave the go-ahead to Idhifa as a treatment for adults with relapsed or refractory acute myeloid leukemia (AML) who have an isocitrate dehydrogenase-2 (IDH2) mutation, which constitutes about 8% to 19% of the AML population, according to Celgene. The decision came about a month ahead of the drug's Aug. 30 user fee action date.
- While Celgene shareholders barely reacted to the news, Agios Pharmaceuticals Inc., which partnered on Idhifa development, saw its shares rise more than 4% to $58.38 apiece in Thursday afternoon trading.
Dive Insight:
Investors have had a love-hate relationship with Celgene's portfolio.
The first half of that relationship stems from the company's strong revenues. In the second quarter, net product sales and total revenue were both up 19% year-over-year, clocking in at $3.26 billion and $3.27 billion, respectively.
The latter half, however, is rooted in Revlimid (lenalidomide), the drug for mantle cell lymphoma and multiple myeloma that, between April and June, raked in more than $2 billion and experienced a 20% growth in sales from the same period in 2016.
Revlimid consistently makes up about 60% of Celgene's revenue. And while the drugmaker doesn't have to worry about patents for the blockbuster therapy running out anytime soon, it has faced pressure to puts its eggs in more than one basket.
In response, Celgene has turned to dealmaking to flesh out its product offerings and pipeline assets. It recently inked a deal with Chinese pharmaceutical company Beigene that focuses on developing treatments for solid tumors, and noted during its latest quarterly earnings call that it plans to build on that agreement. Executives on the call also signaled interest in the emerging class of oncology drugs known as PARP inhibitors.
Idhifa (enasidenib) furthers the diversification cause as well, extending Celgene's cancer footprint into AML.
Elsewhere, the big biotech is working to grow its arsenal of marketed autoimmune disease treatments past Otezla (apremilast), a medicine for psoriatic arthritis and plaque psoriasis. Early in 2017, Celgene got the exclusive right to acquire Swiss biotech Anokion, which is crafting autoimmune therapies that operate via tolerance mechanisms.
More specifically, multiple sclerosis has been a focus for Celgene. The drugmaker plans to submit its experimental medicine ozanmiod to U.S. regulators for approval later this year after Phase 3 results released in May showed the drug significantly reduced patients' annualized relapse rates.
As for Idhifa, its approval hinged on an open-label, single-arm Phase 1/2 study of relapsed or refractory AML patients with the IDH2 mutation. Study participants demonstrated a complete response or complete response with partial hematologic improvement rate of 23%. Idhifa carries a box warning for a potentially fatal condition called differentiation syndrome.
While the drug's market clearance gives Celgene another product to beef up its portfolio, it also allows Agios to shed the clinical-stage title.
"The FDA approval of Idhifa just four years after entering the clinic is the first of what we expect to be multiple first-in-class precision medicines for patients with cancer and rare genetic diseases from our productive discovery engine," Agios CEO David Schenkein said in an Aug. 1 statement. "We look forward to working closely with Celgene to co-commercialize Idhifa and provide access for patients in the U.S. with this devastating disease."
