- Swedish pharmaceutical company Sobi has a lot to be thankful for this week, as the Food and Drug Administration approved its medicine for a rare inflammation disorder based on data that was announced Tuesday as a late-breaking abstract for the American Society of Hematology's upcoming conference.
- The Phase 2/3 study enrolled 34 patients with primary hemophagocytic lymphohistiocytosis (HLH), 27 of whom had refractory, recurrent or progressive disease, or were intolerant to conventional therapy. Results showed that 17, or 63%, of those 27 patients achieved either a complete response, a partial response or HLH improvement when receiving Sobi's emapalumab along with dexamethasone.
- As such, regulators approved emapalumab — now branded as Gamifant — for primary HLH patients who failed conventional therapy. Rami Levin, president of the Sobi's North America business, told BioPharma Dive the biotech has built up a sales team consisting of 30 field representatives and 14 medical science liaisons. Executives estimate there are about 450 physicians who are potential treaters of primary HLH patients.
Sobi has been pursuing M&A to expand its pipeline and portfolio. The first concrete step toward that goal came in July, when the biotech put about $450 million on the line for exclusive global rights to emapalumab, which Novimmune was developing at the time.
Less than four months later, the bet appears to have paid off.
"If I had to dream of the ideal first acquisition, emapalumab would have been that deal," Levin told BioPharma Dive in an interview. He explained the drug checks all the boxes for Sobi's larger strategy, in that it targets a rare disease, would be the first and only therapy approved for that disease, and was in pre-launch phase.
Sobi expects Gamifant to become available in U.S. treatment centers in the first quarter of 2019. Levin noted that, given the quick turnaround from acquiring emapalumab to rolling it out, executives have had to work quickly to get launch preparations in place.
One thing still left to iron out is pricing.
"What will trigger the final decision on the price is less the product approval but more the product availability, and product availability is expected in Q1," Levin said. "We are in constant conversations and discussions with the payers. There's a good collaboration; there's a very clear understanding of the criticality of the disease and the importance of the product. So the discussions are going well, but we haven't yet finalized the process."
If it registers with payers and prescribers, Gamifant should further fuel the growth seen at Sobi in recent quarters. The biotech recorded total revenue of 6.57 billion Swedish krona (around $725 million) over the first nine months of 2018, a year-over-year increase of 42% compared to the same period in 2017.
Gamifant works by binding to and neutralizing a cytokine called interferon gamma, which affects inflammation. Sobi is exploring the drug's use in other diseases where interferon gamma levels are high, such as graft-versus-host disease and secondary HLH.
"As we understand the benefit-risk profile over time, as we expose more patients to it, it will give us a better understanding of where the next opportunities are," Carol Satler, Sobi's vice president of medical affairs in North America, told BioPharma Dive.
To that point, safety results from the Phase 2/3 study found 27% of emapalumab-treated patients experienced mild to moderate infusion-related reactions. Other common adverse reactions were infections, which occurred in more than half of the patients, hypertension and fever.
One patient had an infection caused by pathogens potentially favored by interferon gamma neutralization while receiving emapalumab, but that case resolved with "appropriate treatment," according to the ASH late-breaker.