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FDA approves first drug for anemia tied to rare blood disorder

The Food and Drug Administration on Friday approved the first drug for treating anemia in adults suffering from beta-thalassemia, a rare blood condition that reduces the body’s production of the vital oxygen-carrying protein hemoglobin. Developed by Celgene and biotech partner Acceleron Pharma, the newly named Reblozyl is one of five drugs that attracted Bristol-Myers Squibb in its $74 billion buyout of Celgene earlier this year. The large biotech secured approval of another, Inrebic for myelofibrosis, in August. Patients with severe forms of beta-thalassemia must receive regular blood transfusions for life, a burdensome treatment regimen that comes with the risk of iron overload. In testing, treatment with Reblozyl cut by a third the number of transfusions in about a fifth of patients, compared to only 4.5% of those given placebo.

With Friday’s approval, Celgene’s pipeline passed the second test set out in Bristol-Myers Squibb’s acquisition of the company.

In selling investors on the deal, Bristol-Myers’ management pointed to five Celgene drugs they argued would become the future of the combined company’s business. Executives predicted that group, together with an experimental therapy from Bristol-Myers’ own labs, could earn as much as $15 billion in peak annual sales.

Reblozyl (luspatercept) and Inrebic (fedratinib) were viewed as likely to garner Food and Drug Administration approval. Less certain, however, is the fate of the three other Celgene drugs: ozanimod for multiple sclerosis, liso-cel for lymphoma and ide-cel for multiple myeloma.

To hedge against the risk of regulatory surprises, Bristol-Myers structured its deal to include a contingent value right worth $9 per Celgene share. For that to pay out, all three drugs need to win U.S. approval by predetermined dates next year and in 2021.

So while Reblozyl’s approval is a looked-to milestone, clearance of the three other drugs is more critical for Celgene shareholders.

Six pipeline assets crucial to Bristol-Myers’ deal for Celgene
Drug candidate Lead indication Next step: Previous Celgene peak sales forecast
Inrebic Myelofibrosis Approved $1 billion
ozanimod Multiple sclerosis Filed with FDA, approval expected in 2020 $4 billion to $6 billion
Reblozyl Beta-thalassemia Approved >$2 billion
liso-cel (JCAR017) Lymphoma Data due at ASH, approval expected in 2020 $3 billion
ide-cel (bb2121) Multiple myeloma Approval expected in 2020 or early 2021 >$2 billion
TYK2 Psoriasis Phase 3 readouts in 2020 N/A
TOTAL >$15 billion

*TYK2 inhibitor is from Bristol-Myers’ pipeline; estimate of $15 billion plus in total peak revenue is from Bristol-Myers and Celgene SOURCE: Company presentations

For Acceleron, Reblozyl is the company’s first drug to win U.S. approval, and its OK triggers a $35 million payment from Celgene. Shares in the company rose by 6% in Friday trading.

Reblozyl will be available commercially in one week and will cost $3,441 per 25 mg vial at list price, the companies said. The drug is dosed by weight, typically at a dose of 1 milligram per kilogram.

For the recommended once per every three weeks regimen, that works out to an annual cost over $170,000 — “significantly higher” than expected, according to SVB Leerink analyst Geoffrey Porges.

While a small number of patients treated with Reblozyl experienced thromboembolic events, and 10% reported hypertension, the drug does not come with a black box warning.

The FDA is also reviewing Reblozyl for approval in anemia related to myelodysplastic syndromes, a group of cancer-like bone marrow disorders, and a decision is expected from the agency by April 2020.

Between the two, the companies believe the drug could eventually earn as much as $2 billion a year.

If that comes to pass, Reblozyl will have been good value for Celgene’s money. The large biotech paid $25 million in an initial licensing fee in 2011 and, as of September 30, has paid Acceleron $144 million. (Celgene has funded all development costs for the drug since 2013, however.)

Beta-thalassemia is newly a target for drugmakers. In June, a gene therapy developed by Bluebird bio was approved to treat patients with the condition who are dependent on transfusions. Other companies, like CRISPR Therapeutics, are developing gene editing approaches for the disease as well.