Scilex Holding Company Announces FDA Has Approved Our Request for the Orphan Drug Designation for Colchicine for the Treatment of Pericarditis
Scilex Holding Company (NASDAQ: SCLX) has received FDA approval for Orphan Drug Designation for colchicine in treating pericarditis. Their product Gloperba®, the first and only liquid oral colchicine, is currently indicated for gout flare prophylaxis in adults.
Gloperba® is administered like cough syrup with a dosage of 0.6mg per 5ml, offering advantages for patients who struggle with pills. The liquid format enables more flexible dosing and titration options, particularly beneficial for gout patients with renal or hepatic impairment.
The Orphan Drug Designation applies to conditions affecting fewer than 200,000 individuals in the US. If approved, it would grant seven years of market exclusivity for treating pericarditis, an inflammatory condition of the heart's protective membrane.
Scilex Holding Company (NASDAQ: SCLX) ha ottenuto l'approvazione dalla FDA per la Designazione di Farmaco Orfano per la colchicina nel trattamento della pericardite. Il loro prodotto Gloperba®, la prima e unica colchicina orale liquida, è attualmente indicato per la profilassi delle crisi di gotta negli adulti.
Gloperba® si somministra come uno sciroppo per la tosse con un dosaggio di 0,6 mg ogni 5 ml, offrendo vantaggi ai pazienti che hanno difficoltà a deglutire compresse. Il formato liquido consente una dose più flessibile e opzioni di titolazione, particolarmente utili per i pazienti con gotta che presentano insufficienza renale o epatica.
La Designazione di Farmaco Orfano riguarda condizioni che colpiscono meno di 200.000 persone negli Stati Uniti. Se approvata, garantirebbe sette anni di esclusiva di mercato per il trattamento della pericardite, una condizione infiammatoria della membrana protettiva del cuore.
Scilex Holding Company (NASDAQ: SCLX) ha recibido la aprobación de la FDA para la Designación de Medicamento Huérfano para colchicina en el tratamiento de la pericarditis. Su producto Gloperba®, la primera y única colchicina oral líquida, está actualmente indicado para la profilaxis de ataques de gota en adultos.
Gloperba® se administra como un jarabe para la tos con una dosis de 0,6 mg por cada 5 ml, ofreciendo ventajas para pacientes que tienen dificultad para tragar pastillas. El formato líquido permite una dosificación más flexible y opciones de titulación, especialmente beneficiosas para pacientes con gota que presentan insuficiencia renal o hepática.
La Designación de Medicamento Huérfano se aplica a condiciones que afectan a menos de 200,000 personas en EE.UU. Si se aprueba, otorgaría siete años de exclusividad en el mercado para el tratamiento de la pericarditis, una inflamación de la membrana protectora del corazón.
Scilex Holding Company (NASDAQ: SCLX)가 심낭염 치료를 위한 콜히친에 대해 희귀의약품 지정(FDA) 승인을 받았습니다. 그들의 제품 Gloperba®는 최초이자 유일한 액상 경구용 콜히친으로, 현재 성인의 통풍 발작 예방에 사용되고 있습니다.
Gloperba®는 기침 시럽처럼 5ml당 0.6mg 용량으로 투여되며, 알약 복용이 어려운 환자들에게 유리합니다. 액상 형태는 투약량 조절과 증량이 더 유연하여 신장 또는 간 기능 장애가 있는 통풍 환자에게 특히 유익합니다.
희귀의약품 지정은 미국 내 20만 명 미만의 환자에게 영향을 미치는 질환에 적용됩니다. 승인될 경우, 심낭염 치료에 대해 7년간 시장 독점권이 부여됩니다. 심낭염은 심장을 보호하는 막의 염증성 질환입니다.
Scilex Holding Company (NASDAQ: SCLX) a obtenu l'approbation de la FDA pour la désignation de médicament orphelin concernant la colchicine dans le traitement de la péricardite. Leur produit Gloperba®, la première et unique colchicine orale liquide, est actuellement indiqué pour la prophylaxie des crises de goutte chez l'adulte.
Gloperba® est administré comme un sirop contre la toux, avec un dosage de 0,6 mg pour 5 ml, offrant des avantages aux patients ayant des difficultés à avaler des comprimés. Le format liquide permet une posologie plus flexible et des options de titration, particulièrement bénéfiques pour les patients goutteux souffrant d’insuffisance rénale ou hépatique.
La désignation de médicament orphelin s’applique aux maladies touchant moins de 200 000 personnes aux États-Unis. En cas d’approbation, elle offrirait sept ans d’exclusivité commerciale pour le traitement de la péricardite, une inflammation de la membrane protectrice du cœur.
Scilex Holding Company (NASDAQ: SCLX) hat von der FDA die Zulassung für die Orphan-Drug-Designation für Colchicin zur Behandlung von Perikarditis erhalten. Ihr Produkt Gloperba®, das erste und einzige flüssige orale Colchicin, ist derzeit für die Prophylaxe von Gichtanfällen bei Erwachsenen zugelassen.
Gloperba® wird wie Hustensaft mit einer Dosierung von 0,6 mg pro 5 ml verabreicht und bietet Vorteile für Patienten, die Schwierigkeiten mit Tabletten haben. Das flüssige Format ermöglicht eine flexiblere Dosierung und Titration, was besonders für Gichtpatienten mit Nieren- oder Leberfunktionsstörungen von Vorteil ist.
Die Orphan-Drug-Designation gilt für Erkrankungen, die weniger als 200.000 Personen in den USA betreffen. Bei Zulassung würde dies sieben Jahre Marktexklusivität für die Behandlung der Perikarditis gewähren, einer entzündlichen Erkrankung der schützenden Herzmembran.
- FDA granted Orphan Drug Designation for colchicine in pericarditis treatment
- Potential 7-year market exclusivity if approved for pericarditis
- First and only liquid colchicine formulation in the market
- Enhanced dosing flexibility for patients with kidney/liver impairment
- Approval for pericarditis treatment not yet obtained, only received orphan designation
- market size due to rare disease status (<200,000 patients)
- Development costs may not be recovered from US sales alone
Insights
The FDA's Orphan Drug Designation for Scilex's colchicine (Gloperba®) for pericarditis represents a significant regulatory milestone with several important implications.
This designation acknowledges pericarditis as a rare disease affecting fewer than 200,000 Americans and grants Scilex valuable benefits including potential 7-year market exclusivity upon approval, tax credits for clinical trials, and exemption from FDA application fees. These incentives substantially improve the economics of developing this treatment.
It's critical to understand this is not FDA approval for treating pericarditis. Scilex must still complete clinical trials and submit data demonstrating safety and efficacy before Gloperba can be marketed for this indication. This designation is best viewed as reducing regulatory hurdles and enhancing commercial potential rather than immediate revenue generation.
Gloperba's liquid formulation offers distinct advantages over traditional tablet forms of colchicine, including more precise dosing adjustments and easier administration for patients who struggle with pills. For pericarditis patients with renal or hepatic impairment, this formulation may also enable better dose optimization to reduce side effects.
This regulatory win aligns with Scilex's strategy to maximize the potential of its existing products across multiple indications while maintaining focus on non-opioid treatments. The company appears to be executing a thoughtful portfolio expansion approach by leveraging its approved products into additional therapeutic applications where development costs and regulatory risks may be lower than entirely new compounds.
- Gloperba® is the first and only liquid oral version of the anti-gout medicine colchicine indicated for the prophylaxis of painful gout flares in adults.
- Pericarditis is an inflammation of the pericardium, the sac-like membrane that surrounds and protects the heart. It occurs when the pericardium becomes irritated or infected, leading to swelling and pain.
PALO ALTO, Calif., April 16, 2025 (GLOBE NEWSWIRE) -- Scilex Holding Company (Nasdaq: SCLX, “Scilex” or “Company”), an innovative revenue-generating company focused on acquiring, developing and commercializing non-opioid pain management products for the treatment of acute and chronic pain, following the formation of its proposed joint venture with IPMC Company, neurodegenerative and cardiometabolic disease, today announced that FDA has approved our request for the Orphan drug designation for colchicine for the treatment of pericarditis.
Gloperba® is taken orally like cough syrup. The dosage of 0.6mg per 5ml (teaspoon) can fill that important void in treatment where patients may have difficulty swallowing pills. It can also provide more adjustable dosing, titration and dose-reduction options in specific populations, especially for gout patients with renal or hepatic impairment and reduce side effects with the goal to improve patient convenience and disease management.
Under the Orphan Drug Act, the FDA may grant orphan designation to a drug or biological product intended to treat a rare disease or condition, which is generally a disease or condition that affects fewer than 200,000 individuals in the United States, or more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making the product available in the United States for this type of disease or condition will be recovered from sales of the product. Orphan drug designation must be requested before submitting an NDA. After the FDA grants orphan drug designation, the identity of the therapeutic agent and its potential orphan use are disclosed publicly by the FDA. Orphan drug designation does not convey any advantage in or shorten the duration of the regulatory review and approval process. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan drug exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same indication for seven years from the date of such approval, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity by means of greater effectiveness, greater safety or providing a major contribution to patient care or in instances of drug supply issues.
For more information on Scilex Holding Company, refer to www.scilexholding.com
For more information on Semnur Pharmaceuticals, Inc., refer to www.semnurpharma.com
For more information on ZTlido® including Full Prescribing Information, refer to www.ztlido.com
For more information on ELYXYB®, including Full Prescribing Information, refer to www.elyxyb.com
For more information on Gloperba®, including Full Prescribing Information, refer to www.gloperba.com
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About Scilex Holding Company
Scilex Holding Company is an innovative revenue-generating company focused on acquiring, developing and commercializing non-opioid pain management products for the treatment of acute and chronic pain and, following the formation of its proposed joint venture with IPMC Company, in neurodegenerative and cardiometabolic disease. Scilex targets indications with high unmet needs and large market opportunities with non-opioid therapies for the treatment of patients with acute and chronic pain and is dedicated to advancing and improving patient outcomes. Scilex’s commercial products include: (i) ZTlido® (lidocaine topical system)
In addition, Scilex has three product candidates: (i) SP-102 (10 mg, dexamethasone sodium phosphate viscous gel) (“SEMDEXA™” or “SP-102”), a novel, viscous gel formulation of a widely used corticosteroid for epidural injections to treat lumbosacral radicular pain, or sciatica, for which Scilex has completed a Phase 3 study and was granted Fast Track status from the FDA in 2017; (ii) SP-103 (lidocaine topical system)
Scilex Holding Company is headquartered in Palo Alto, California.
About Semnur Pharmaceuticals, Inc.
Semnur Pharmaceuticals, Inc. (“Semnur”), a wholly-owned subsidiary of Scilex, is a clinical late-stage specialty pharmaceutical company focused on the development and commercialization of novel non-opioid pain therapies. Semnur’s product candidate, SP-102 (SEMDEXA™), is the first non-opioid novel gel formulation administered epidurally in development for patients with moderate to severe chronic radicular pain/sciatica.
Semnur Pharmaceuticals, Inc. is headquartered in Palo Alto, California
Forward-Looking Statements
This press release and any statements made for and during any presentation or meeting concerning the matters discussed in this press release contain forward-looking statements related to Scilex and its subsidiaries under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the granting of Orphan drug designation status by the FDA, the estimates for the obesity drug treatment and Alzheimer’s global drug market and the global spending figures, KDS2010’s potential to demonstrate a more favorable efficacy profile compared to existing obesity treatments, Scilex Bio JV’s ability to develop and commercialize treatments for obesity, neurodegenerative, and cardiometabolic disease.
Risks and uncertainties that could cause Scilex’s actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: Scilex’s ability to gain market acceptance of KDS2010; Scilex’s ability to develop and commercialize treatments for obesity, neurodegenerative, or cardiometabolic disease; risks associated with the unpredictability of trading markets and whether a market will be established for Scilex’s common stock; general economic, political and business conditions; the risk that the potential product candidates that Scilex develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Scilex’s product candidates; the risk that Scilex will be unable to successfully market or gain market acceptance of its product candidates; the risk that Scilex’s product candidates may not be beneficial to patients or successfully commercialized; the risk that Scilex has overestimated the size of the target patient population, their willingness to try new therapies and the willingness of physicians to prescribe these therapies; risks that the outcome of the trials and studies for SP-102, SP-103 or SP-104 may not be successful or reflect positive outcomes; risks that the prior results of the clinical and investigator-initiated trials of SP-102 (SEMDEXA™), SP-103 or SP-104 may not be replicated; regulatory and intellectual property risks; and other risks and uncertainties indicated from time to time and other risks described in Scilex’s most recent periodic reports filed with the Securities and Exchange Commission, including Scilex’s Annual Report on Form 10-K for the year ended December 31, 2024 and subsequent Quarterly Reports on Form 10-Q that the Company has filed or may file with the SEC, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and Scilex undertakes no obligation to update any forward-looking statement in this press release except as may be required by law.
Contacts:
Investors and Media
Scilex Holding Company
960 San Antonio Road
Palo Alto, CA 94303
Office: (650) 516-4310
Email: investorrelations@scilexholding.com
Website: www.scilexholding.com
References
- Comorbidities of Gout and Hyperuricemia in the US General Population: NHANES 2007-2008
- Stewart et al. Arthritis Research & Therapy (2020) 22:28; https://doi.org/10.1186/s13075-020-2120-7
- https://www.sciencedirect.com/science/article/pii/S0049017220301220
- https://pmc.ncbi.nlm.nih.gov/articles/PMC10285625/
SEMDEXA™ (SP-102) is a trademark owned by Semnur Pharmaceuticals, Inc., a wholly-owned subsidiary of Scilex Holding Company. A proprietary name review by the FDA is planned.
ZTlido® is a registered trademark owned by Scilex Pharmaceuticals Inc., a wholly-owned subsidiary of Scilex Holding Company.
Gloperba® is the subject of an exclusive, transferable license to use the registered trademark by Scilex Holding Company.
ELYXYB® is a registered trademark owned by Scilex Holding Company.
All other trademarks are the property of their respective owners.
© 2025 Scilex Holding Company All Rights Reserved.
FAQ
What benefits does the FDA Orphan Drug Designation provide SCLX for Gloperba?
How is SCLX's Gloperba different from traditional colchicine medications?
What market opportunity does pericarditis present for SCLX's Gloperba?
What advantages does Gloperba offer for patients with renal or hepatic impairment?
