- Alexion Pharmaceuticals is trying to get a speedy approval of its Soliris follow-on drug, announcing Tuesday it used a valuable fast pass that shaves down the regulatory review time by several months.
- Though it normally takes about a year for the Food and Drug Administration to clear or reject a Biologics Licensing Application (BLA), the agency should make a decision on the one submitted for ALXN1210 in the next eight months because it came equipped with a priority review voucher.
- The FDA awards PRVs to companies that bring treatments for either rare pediatric illnesses or tropical diseases to market. Alexion has kept two in its back pocket since the end of 2015, and still holds onto the other, according to a spokesperson.
Soliris (eculizumab) is one of the most expensive drugs in the world — and is unsurprisingly highly lucrative. First approved in 2007 for a rare blood disorder called paroxysmal nocturnal hemoglobinuria, Soliris has since raked in more than $15 billion for Alexion.
While Soliris supplies roughly 90% of the biotech's net product sales, one thing the drug hasn't drummed up is a PRV. The two in Alexion's arsenal instead came from its other marketed products, Strensiq (asfotase alfa) and Kanuma (sebelipase alfa), both of which notched FDA OKs in 2015. Strensiq treats perinatal/infantile- and juvenile-onset hypophosphatasia, while Kanuma is for lysosomal acid lipase deficiency.
One of those vouchers — it's unclear which — is now attached to the BLA for ALXN1210, known also as ravulizumab. Alexion is looking to get the drug approved in PNH as well, and submitted it to regulators right on the heels of a positive data presentation at the European Hematology Association's annual meeting.
At the meeting, which wrapped up Sunday, late-stage clinical data showed that ALXN1210 was faster than Soliris at normalizing lactate dehydrogenase levels, which indicates less damage to red blood cells. The investigational therapy was also better at inhibiting a protein that promotes inflammatory responses and cell destruction.
On the former measure, the results demonstrated that, after 22 days of treatment, a greater portion of patients taking ravulizumab versus Soliris had normalized lactate dehydrogenase levels at all but one of thirteen time points.
"After this presentation it's clear to us that '1210 is even more differentiated than we had anticipated ... and is likely to dominate treatment of newly diagnosed PNH," Leerink analyst Geoffrey Porges wrote in a June 18 note.
"We also find this data supportive of our expectation that ravulizumab will achieve a majority overall share in this indication, through both capturing new patients and through switches of established Soliris patients," he wrote.
Leerink estimates combined sales of Soliris and ALXN1210 will hit $5.2 billion in 2022, and will then grow by low single-digit percentages to reach $5.6 billion in 2025
Alexion clearly sees the value in these drugs as well. It recently secured a label expansion for Soliris as well as three new U.S. patents that extend the product's exclusivity to 2027. As for ALXN1210, the use of a PRV — which can be sold for hundreds of millions of dollars — signals just how profitable Alexion thinks its new offering can be.