Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for VCA-894A, an investigational antisense oligonucleotide therapy intended to treat Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). The designation was issued by the FDA's Office of Orphan Products Development together with the Office of Pediatric Therapeutics.

VCA-894A targets a specific patient first identified at a young age with the rare CMT2S subtype of Charcot-Marie-Tooth disease. This inherited neuromuscular condition gradually produces muscle weakness and diminished motor function, with worldwide prevalence estimated below one in one million individuals. VCA-894A focuses on a distinctive CMT2S variant not previously documented in other patients. Disease severity and presentation vary according to the range of genetic changes linked to CMT disorders.

The FDA concluded that CMT2S meets rare pediatric disease criteria because it represents a serious or life-threatening disorder whose primary effects occur in individuals from birth through age 18 and satisfies the statutory definition of rarity.

"CMT2S presents a profound inherited neuropathy with few available treatment choices for patients and families," stated Mihael H. Polymeropoulos, M.D., President, Chief Executive Officer and Chairman of Vanda. "The FDA's Rare Pediatric Disease Designation highlights the substantial unmet medical need within this group and bolsters our work to create a potentially transformative option for those impacted by this uncommon condition."

CMT2S involves chronic progressive sensory and motor deficits that may culminate in severe disability, including inability to walk. The FDA's designation letter emphasized that the disease's serious effects predominantly impact pediatric patients.

The Rare Pediatric Disease Designation aims to stimulate development of therapies for serious or life-threatening rare conditions that mainly affect children. Sponsors whose products satisfy all statutory criteria may qualify for a priority review voucher upon approval of a qualifying marketing application. Any future voucher eligibility will be assessed during review and approval of the marketing application.

Vanda continues to advance its investigational therapy for CMT2S and will maintain close collaboration with the FDA on the development program.

References:

Smieszek, S. et al. Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S. Mol Ther Nucleic Acids 36, 102479 (2025).
Charcot-Marie-Tooth disease type 2S. Orpha.net (2025).

About Vanda Pharmaceuticals Inc.

Vanda is a leading global biopharmaceutical company dedicated to discovering and commercializing innovative therapies that address significant unmet medical needs and enhance patient outcomes. Additional information is available at www.vandapharma.com and on X at @vandapharma.

About Charcot-Marie-Tooth Disease Type 2S (CMT2S)

Charcot-Marie-Tooth disease type 2S is a rare inherited neurological disorder marked by progressive degeneration of peripheral nerves that produces sensory and motor impairment. Over time the condition can result in substantial functional limitations and disability.

About VCA-894A

VCA-894A is a 2'-O-methoxyethyl (MOE) phosphorothioate oligonucleotide sodium salt. It specifically targets a cryptic splice site variant in IGHMBP2 that underlies CMT2S. Antisense oligonucleotides may offer broad utility across nervous system and systemic disorders.

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

Various statements in this press release, including but not limited to statements regarding the estimated prevalence of CMT2S, the therapeutic potential of VCA-894A for patients affected by CMT2S, a sponsor's eligibility to receive a priority review voucher, and Vanda's clinical development and regulatory plans and strategies for VCA-894A, are "forward-looking statements" under the securities laws. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Forward-looking statements are based upon current expectations and assumptions that involve risks, changes in circumstances and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, the accuracy of the estimates of the prevalence of CMT2S worldwide, the efficacy of VCA-894A in the treatment of patients with CMT2S, Vanda's ability to complete the development and obtain regulatory approval of VCA-894A for the treatment of CMT2S, a product's ability to meet the statutory requirements necessary for its sponsor to become eligible to receive a priority review voucher and the ultimate receipt of the voucher, and the ability of Vanda and the FDA to continue to work closely together with regard to the VCA-894A development program. Therefore, no assurance can be given that the results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Forward-looking statements in this press release should be evaluated together with the various risks and uncertainties that affect Vanda's business and market, particularly those identified in the "Cautionary Note Regarding Forward-Looking Statements", "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's most recent Annual Report on Form 10-K, as updated by Vanda's subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the U.S. Securities and Exchange Commission, which are available at www.sec.gov.

All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this press release is provided only as of the date of this press release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Corporate Contact:

Kevin Moran
Senior Vice President, Chief Financial Officer and Treasurer
Vanda Pharmaceuticals Inc.
202-734-3400
[email protected]

Jim Golden / Jack Kelleher / Dan Moore
Collected Strategies
[email protected]

SOURCE Vanda Pharmaceuticals Inc.