RARE Daily

FDA Grants Rare Pediatric Disease Designation for Lactiga’s Therapy for CVID

August 14, 2023

Rare Daily Staff


The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation for Lactiga’s biologic, LCTG-001, a mucosal antibody replacement therapy for patients with common variable immunodeficiency.


Common variable immunodeficiency (CVID) is a chronic and incurable heterogeneous immune disorder characterized by a decline in antibodies resulting in a weakened immune system and increased susceptibility to infections. Patients who suffer from CVID experience a decreased quality of life and increased risk of premature mortality compared to the general population. In its letter granting RPD designation, the FDA stated there is “sufficient information to demonstrate that increased mortality rate, developmental delay, and failure to thrive are serious or life-threatening manifestations that primarily affect children [with CVID].”


LCTG-001 is a mucosal antibody replacement therapy comprised of polyclonal Immunoglobulin A (IgA) for nasal administration and is on course to potentially be the first FDA-approved biologic from human milk.

The FDA’s Rare Pediatric Disease designation program is intended to encourage and accelerate innovative therapies to treat rare, life-threatening diseases that primarily affect individuals aged from birth to 18 years. By obtaining this designation, Lactiga has access to valuable incentives and support from the FDA during its development program. Upon approval of LCTG-001, Lactiga qualifies to receive a priority review voucher for any subsequent marketing application which can be sold or transferred to other companies. Most recently, Sarepta Therapeutics sold a voucher for $102 million.

“This is the start of Lactiga’s ambitious mission to shift the paradigm of therapeutic antibody development from systemic to mucosal delivery, where many pathogens initially enter the body,” said Rikin Mehta, Lactiga co-founder and CEO. “By targeting the mucosal immune system, we can create more effective therapies that prevent infection at the point of entry, leading to a much better quality of life, especially in our most vulnerable populations.”



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