SNY's Rare Disease Drug Efdoralprin Alfa Gets EMA's Orphan Drug Tag

Sanofi SNY announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (“ODD”) to efdoralprin alfa (SAR447537, formerly known as INBRX-101), an investigational biologic drug for the treatment of alpha-1 antitrypsin deficiency (AATD)–related emphysema in adult patients.

The ODD status is granted by the EMA to promote new drugs or indications for existing drugs to treat, prevent, or diagnose rare, life-threatening, or debilitating medical diseases or conditions affecting a small percentage of the population (less than 5 in 10,000 individuals).

AATD is a rare, inherited lung and liver disease marked by low levels of alpha-1 antitrypsin (AAT) protein produced by the liver. It affects lung function, resulting in lung damage and the development of COPD, including emphysema. Patients severely infected by the disease may sometimes need lung transplantation. Efdoralprin alfa is a recombinant human AAT protein fused to the Fc portion of a human antibody. It works by inhibiting neutrophil elastases, an enzyme responsible for lung tissue damage in AATD patients.

Efdoralprin alfa, currently in mid-stage development, was added to Sanofi’s rare disease pipeline following the acquisition of Inhibrx in 2024.

Over the past year, SNY’s shares have risen 1.3% compared with the industry’s 13.8% rise.

Efdoralprin Alfa ODD Based on ElevAATe Study Data

The ODD was based on positive data from the global phase II ElevAATe study, which demonstrated that treatment with efdoralprin alfa administered every three weeks (Q3W) or four weeks (Q4W) resulted in a statistically significant greater mean increase in functional AAT levels — within the normal range — based on trough concentrations at steady state, compared with weekly plasma-derived augmentation therapy at week 32.

Treatment with efdoralprin alfa also led to a greater mean increase in average functional AAT (fAAT) concentrations and a higher percentage of days with levels above the lower limit of the normal range for both Q3W and Q4W dosing regimens, thereby meeting the study’s key secondary endpoints.

Efdoralprin alfa had earlier earned Fast Track and Orphan Drug designations from the FDA for the treatment of AATD emphysema.

SNY’s Zacks Rank & Stocks to Consider

SNY currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are ANI Pharmaceuticals ANIP, CorMedix CRMD and Castle Biosciences CSTL. ANIP, CRMD and CSTL currently sport a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share (EPS) have increased from $7.29 to $7.56. Over the same period, EPS estimates for 2026 have surged from $7.81 to $8.08. Shares of ANIP have surged 51.8% in the past year.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 21.24%.

Over the past 60 days, estimates for CorMedix’s 2025 EPS have increased from $1.85 to $2.87, while 2026 EPS estimates have risen from $2.49 to $2.88 over the same period. Shares of CRMD have surged 29.1% over the past year.

CorMedix’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 27.04%.

Over the past 60 days, the loss estimate for Castle Biosciences has narrowed from 64 cents to 34 cents in 2025. Over the same period, loss estimates for 2026 have improved from $1.82 to $1.06. CSTL stock has rallied 46.4% over the past year.

Castle Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining quarter, with the average surprise being 66.11%.

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This article originally published on Zacks Investment Research (zacks.com).

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