BioArctic’s investigational MSA therapy gains FDA recognition

SWEDEN—BioArctic AB, a Swedish biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted exidavnemab, its investigational drug for treating multiple system atrophy (MSA), Orphan Drug Designation (ODD).

This designation is a significant milestone, as it provides incentives to encourage the development of treatments for rare diseases with high unmet medical needs.

MSA is a rare, rapidly progressive, and fatal neurological disorder that affects the central and autonomic nervous systems.

It is characterized by the pathological accumulation of alpha-synuclein protein in the brain, which damages nerve cells.

This leads to severe symptoms such as impaired balance, movement difficulties, and dysfunction in basic autonomic processes like breathing, digestion, and bladder control.

Currently, there is no cure or treatment available to slow the progression of MSA.

Exidavnemab is being developed as a novel disease-modifying therapy targeting synucleinopathies such as MSA and Parkinson’s disease.

It is a monoclonal antibody (mAb) designed to selectively bind to soluble alpha-synuclein aggregates, specifically oligomers and protofibrils, which are thought to play a central role in disease progression.

Exidavnemab aims to reduce the harmful effects of these aggregates by facilitating their clearance, potentially preserving neuronal function and slowing disease progression.

The FDA’s Orphan Drug Designation program supports the development of treatments for diseases affecting fewer than 200,000 people in the United States.

Drugs granted this status are eligible for several benefits, including tax credits for clinical trials, exemption from certain FDA fees, and up to seven years of market exclusivity following approval.

These incentives significantly reduce the financial burden of drug development while enhancing the commercial potential of therapies targeting rare conditions.

While this designation highlights the critical need for innovative treatments like exidavnemab, it does not guarantee clinical success or FDA approval.

The drug remains in an investigational phase, and its safety and efficacy are yet to be established through rigorous clinical trials.

BioArctic’s achievement underscores its commitment to addressing pressing medical challenges associated with rare neurodegenerative diseases.

The company’s focus on MSA also aligns with its broader strategy of developing therapies for synucleinopathies, which could have implications beyond MSA, including Parkinson’s disease.

This regulatory milestone provides a clearer pathway for exidavnemab’s development while drawing attention to the urgent need for effective treatments for devastating conditions like MSA.

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