Biogen and Swedish Orphan fixed for haemophilia success

Today’s positive data for one of their long-acting haemophilia treatments, rFIXFc, mean that Biogen Idec and Swedish Orphan Biovitrum will now need pretty big rear-view mirrors to see the competition coming up behind them.

Investors also clearly thought the result in the smaller haemophila B indication, which holds out the tantalising prospect of fortnightly dosing for some patients, could provide a positive readthrough to the much larger and more significant A-Long haemophilia A trial. Shares in Swedish Orphan were trading up 44% on the news at SKr37.40, their highest level for more than 30 months, while Biogen stock rose by 2% to $155.81.

The positive results

The 123-patient trial of males aged 12 and older showed that rFIXFc managed to control bleeding effectively, and in one of the treatment arms, where patients were allowed to tailor their dosing to maintain factor IX levels of between 1% and 3%, more than half were able to cut transfusions down to at least once every 14 days. Usually even patients with mild symptoms have to get transfusions every three to four days. 

Importantly, during the study no inhibitors were detected, signalling that no patients were developing an immune response to the new factor. rFIXFc also showed an impressive half life of 82 hours, which compares more than favourably against Pfizer’s BeneFIX, which had a 34-hour half life in this trial. Last year BeneFIX, the leading haemophilia A drug, had sales of $693m.

It is hoped that in the A-Long study, which follows a similar dosing regimen, rFVIIIFc will also be able to reduce transfusion rates, although these might only go down to once weekly, given how haemophilia A and B differ. Data for rFVIIIFc are expected in the coming weeks (Event – Biogen's long-acting haemophilia agents near clinical validation, August 14, 2012).

If positive, the trial could be yet another strong catalyst for the shares of both Swedish Orphan and Biogen. Massachusetts-based Biogen has already seen its stock increase by more than 60% since the start of the year.

Approval horizon

US partner Biogen now intends to submit a US BLA in the first half of next year, meaning approval could come by the end of 2013. For Swedish Orphan the wait for European approval might be a bit longer as one of the conditions for marketing authorisation in the region is that it has to complete a study in children younger than 12 before filing.

This could mean that Swedish Orphan's share of the longer-acting factor spoils will not be delivered into its bank account until 2014 at the earliest. Still, they will be welcome revenues and are forecast to come in at $110m by 2018 for rFVIIIFc and $44m for rFIXFc, according to data from EvaluatePharma. Biogen for its part is expected to see 2018 sales of $115m and $61m respectively.

However, judging from today’s share price reaction these figures could be increased, but those expecting a stellar launch trajectory for either product might be wise to cool their jets.

Slow ramp-up

Although the data appear compelling and patients would almost certainly welcome relief from the onerous burden of thrice or twice-weekly infusions, many are expecting a slow ramp-up to sales.

The haemophilia space has seen safety scares from innovative product. Few will have forgotten the impact of inadvertent HIV transmission from the use of blood plasma, and as such physicians might want to wait to see longer-term inhibition formation data and also confirm the efficacy and duration of action of the newer longer-acting factors.

So, while some are forecasting blockbuster status for Biogen and Swedish Orphan, which are not set to see any real competition before 2015 or 2016, it might not be until 2020 that these figures are actually achieved.

To contact the writer of this story, email Lisa Urquhart in London at [email protected]