New Products

FDA hits a green light on 2 more rare disease drugs, giving Alexion an early shot at protecting PNH franchise

Faced with the prospect of a partial government shutdown later today, the FDA has pulled the trigger on a pair of new drug approvals.

Alexion got its nod 2 months early for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris. And Stemline Therapeutics won an FDA approval to market Elzonris (tagraxofusp-erzs) for rare cases of blastic plasmacytoid dendritic cell neoplasm, or BPDCN.

The FDA has now approved 59 new drugs through CDER in 2018, part of its record-setting year as the industry enjoys a surge of new marketing OKs.


Geoffrey Porges

Alexion $ALXN is planning to price Ultomiris at a discount to Soliris, long one of the most expensive therapies in the world. ALXN1210 proved just as effective as their big drug Soliris in treating PNH, with less frequent dosing that will now pave the way to a discount price. Leerink’s Geoffrey Porges counts himself as a supporter of this strategy ahead of prospective competitors for their PNH franchise.

While investors may react negatively to the initial impact of this pricing headwind (revenue for the PNH indication for established patients down 10%), we believe the stock already discounts substantial price erosion and market share loss to competitors. Instead, Alexion’s decision to move toward more sustainable pricing with a superior product should front-run and counter these potential risks, and build toward a more stable revenue trajectory and longevity than currently expected. 

Stemline $STML, meanwhile, had to endure a kerfuffle earlier on regarding its tardy acknowledgement of a patient death during a small study of their drug, but the agency nonetheless providing a thumbs-up on a small set of data.

The drug was waved through after posting widely varying results from two patient cohorts which received the drug in a tiny single-arm study.

The efficacy of Elzonris was studied in two cohorts of patients in a single-arm clinical trial. The first trial cohort enrolled 13 patients with untreated BPDCN, and seven patients (54%) achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). The second cohort included 15 patients with relapsed or refractory BPDCN. One patient achieved CR and one patient achieved CRc.

The OK comes with a black box warning alerting physicians to be on alert for a host of potential side effects, but this is one condition where there are no approved drugs, putting it on a priority list for the FDA, which offered its breakthrough drug designation to help speed it along.


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Biotech Investment Analyst
SV Health Investors Boston, MA
Director, Program Management
Contrafect Corporation New York, NY
Director, Translational Sciences
Cadent Therapeutics Cambridge, MA

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