Spotlight on Ravulizumab 3- developments

Just a few days ago, Alexion reported on its financial performance for 2019. Its net product sales last year were $10 million short of $5 billion. ( Gross Sales income would be about 10% more)

A growth of over $800 million on 2018.

Sales of Alexion’s other products STRENSIQ and KANUMA continued to grow but eculizumab remains by far its biggest seller growing by nearly $400 million in the year. But last year also saw sales  of its newer version Ravulizumab -cwvz . $338.9 millions’ worth. The full year equivalent of maybe 600 plus PNH and some aHUS patients had accessed or transitioned to Ravulizumab.

Although some of those sales were for the treatment of aHUS in the USA , Alexion reported that applications for aHUS licensing in EU, Japan and UK were still pending.

Perhaps a surprise to many was the announcement that in November and December 2019, applications for approval of a new formula version of Ravulizumab  , the 100mg/mL formulation,  to replace the current 300mg/mL vials were submitted in the EU and U.S., respectively.

A higher concentration formulation would reduce the time taken for infusing Ravulizumab from 2 hours ,or so , to 45 minutes. Not much longer than the 35 minutes it takes for eculizumab. Alexion plans to file for regulatory approval of this formulation in Japan in mid-2020.

Alexion also reported that enrolment to a trial of a new way to deliver Ravulizumab, subcutaneously once a week, for PNH and aHUS has been completed. Data from the trial is expected before July. For some there may be a trade between more frequency and controlling self administration.

Other news about Ravulizumab include trials for children and adolescents with PNH and aHUS, for adults with gMG, NMSOD and application to the FDA in December for the treatment of Amyotrophic Lateral Sclerosis ( ALS).

Alexion also is planning an “exploratory” clinical study of Ravulizumab for Primary Progressive Multiple Sclerosis (PPMS)

And lastly as frequent readers of this site might recall from previous articles about aHUS renaming and reclassification into sub categories of TMA’s ( click here for previous article) two other announcements indicate that direction of travel.

 Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA): Alexion plans to initiate limited dose-ranging studies of Ravulizumab in adults and children with HSCT-TMA in the first half of 2020, followed by Phase 3 trials in the second half of 2020, pending regulatory feedback.

Complement Mediated Thrombotic Microangiopathy (CM-TMA): Alexion plans to initiate a Phase 3 study of Ravulizumab in CM-TMA in the second half of 2020, pending regulatory feedback.

As  CM TMA has been synonymous with aHUS ( primary) could this be a venture into all those TMAs  where other factors hamper the operation of Complement e.g. MGUS when Complement itself is not defective. Maybe just using when needed for as long as is needed.

Alexion’s full announcement can be read following the link here.