Alexion files for key drug with priority review voucher
Trust favourite Alexion Pharmaceuticals (Nasdaq: ALXN) yesterday confirmed it had made a US regulatory submission for approval of ALXN1210, its long-acting C5 complement inhibitor, for the treatment of the ultra-rare condition of paroxysmal nocturnal hemoglobinuria (PNH). Alexion is a cornerstone holding for both the OrbiMed-managed Biotech Growth Trust (BIOG, 6.7% of NAV) and Worldwide Healthcare Trust (WWH, 5%), but is also included in Swiss-listed BB Biotech (BBB) (4%) among others.
ALXN1210 is critical to Alexion’s strategy to maintain its dominance in the C5 space, against the threat from potential biosimilars or other copy-type products to its blockbuster Soliris in the 2020s. The company has already this year reported results of two Phase III studies with ALXN1210, which have shown non-inferiority in statistical terms with trends towards superiority for the longer-acting product in comparison with Soliris. A third study of ALXN1210 in atypical hemolytic uremic syndrome (aHUS), the second indication for which Soliris is approved, is due in Q4 this year. ALXN-1210 is dosed every eight weeks versus every two weeks for Soliris, which represents a significant advantage.
The company confirmed it has used a rare disease priority review voucher – which are tradeable and worth around $150m – to obtain an expedited eight-month review by the FDA instead of the standard 12-month review, suggesting approval may come before the year end. This highlights the importance Alexion is placing on getting ALXN-1210 approved quickly and established in the market, well before any possible competitors emerge. Alexion is expects to file ALXN1210 in the EU by mid-year and in Japan in H2.
Potential Solisir biosomilars include Amgen’s ABP959 and Generium’s GNR-045 – although Alexion has recently obtained extended patent coverage to 2027 – while other C5 antibodies (which are not intended to be treated as biosimilar ) include Novartis/MorphoSys tesidolumab (Phase II for PNH), Regenron’s REGN3918 (Phase I) and Roche’s RO7112689/RG6107 (Phase I/II).
Alexion has recently completed its acquisition of Sweden’s Wilson Therapeutics, which will add a late-stage candidate, WTX101, to its R&D pipeline. WTX101 is in Phase III studies for the treatment of Wilson disease, a rare genetic disorder.