Back to top

Image: Bigstock

AstraZeneca's (AZN) Rare Blood Disorder Drug Gets FDA Nod

Read MoreHide Full Article

AstraZeneca (AZN - Free Report) announced that the FDA had approved Voydeya (danicopan) for treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and chronic blood disorder.

Voydeya is a first-in-class oral Factor D inhibitor, which has been approved by the FDA as an add-on to standard-of-care C5 inhibitors, Ultomiris (ravulizumab) or Soliris (eculizumab), for the treatment of extravascular hemolysis in adult patients with PNH. Ultomiris and Soliris are also marketed by AstraZeneca for treating PNH and some other rare disease indications. Voydeya is approved for use in combination with C5 inhibitor therapy for the 10-20% of PNH patients who experience “hemolysis” or destruction of red blood cells despite treatment with such C5 inhibitors.

Voydeya’s approval is based on data from the pivotal ALPHA phase III study. In the study, Voydeya met the primary endpoint of change in hemoglobin from baseline to week 12 and all key secondary endpoints.

In the past year, AstraZeneca’s stock has declined 3.8% against the industry’s 25.8% rise.

Zacks Investment Research
Image Source: Zacks Investment Research

The drug was approved in Japan in January while regulatory applications seeking approval for Voydeya are under review in several countries, including the European Union. In February, the Committee for Medicinal Products for Human Use of the European Medicines Agency gave a positive opinion recommending approval of Voydeya for PNH.

AstraZeneca is also evaluating Voydeya in a phase II study for geographic atrophy.

Voydeya was added to AstraZeneca’s pipeline with the July 2021 acquisition of rare disease drugmaker Alexion. The acquisition added Alexion’s five marketed rare disease products, including Ultomiris and Soliris, and its pipeline of immune-mediated rare disease candidates.

Ultomiris is a key revenue driver in AstraZeneca’s rare disease portfolio. The drug recorded revenues of $2.97 billion in 2023, up 52% at constant exchange rate, driven by growth in neurology indications, geographic expansions in new markets and continued conversion from Soliris. Soliris sales were $3.14 billion in 2023, down 14% year over year.

Zacks Rank and Stocks to Consider

Currently, AstraZeneca has a Zacks Rank #3 (Hold).

Some better-ranked stocks in the healthcare sector are Vanda Pharmaceuticals (VNDA - Free Report) , ADMA Biologics (ADMA - Free Report) and MorphoSys , sporting a Zacks Rank #1 (Strong Buy) each. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, 2024 estimates for Vanda Pharmaceuticals have improved from a loss of 46 cents to earnings of 1 cent. For 2025, loss estimates have narrowed from 94 cents to 48 cents per share in the past 60 days. In the past year, shares of VNDA have declined 39.8%.

Vanda Pharmaceuticals delivered a three-quarter average earnings surprise of 92.88%.

In the past 60 days, estimates for ADMA Biologics’ 2024 earnings per share have improved from 22 cents to 30 cents. Estimates for 2025 have increased from 32 cents to 50 cents. In the past year, shares of ADMA Biologics have risen 97.0%.

Earnings of ADMA Biologics beat estimates in three of the last four quarters while meeting the same once. ADMA delivered a four-quarter average earnings surprise of 85.0%.

In the past 60 days, estimates for MorphoSys’ 2024 loss per share have narrowed from $2.26 to $2.08. Estimates for 2025 have narrowed from a loss of $1.09 per share to a loss of 89 cents per share. In the past year, shares of MOR have risen 358.5%.


See More Zacks Research for These Tickers


Normally $25 each - click below to receive one report FREE:


AstraZeneca PLC (AZN) - free report >>

Vanda Pharmaceuticals Inc. (VNDA) - free report >>

ADMA Biologics Inc (ADMA) - free report >>

Published in