Zevra’s MIPLYFFA™ (arimoclomol) Receives Approval from U.S. Food and Drug Administration for Use in Patients with Niemann-Pick Disease Type C (NPC)
XOMA Royalty announced that Zevra Therapeutics has received FDA approval for MIPLYFFA™ (arimoclomol), the first therapy approved for use in patients with Niemann-Pick Disease Type C (NPC). MIPLYFFA™ is approved for use in combination with miglustat to treat neurological manifestations of NPC in patients two years and older.
XOMA Royalty is entitled to receive a mid-single digit royalty on MIPLYFFA™ sales and up to $52.6 million in milestones. This approval marks MIPLYFFA™ as the sixth commercial asset in XOMA Royalty's portfolio. In June 2023, XOMA Royalty acquired these rights from LadRx for a $5 million upfront payment plus a share of future event-based milestones.
XOMA Royalty ha annunciato che Zevra Therapeutics ha ricevuto l'approvazione della FDA per MIPLYFFA™ (arimoclomol), la prima terapia approvata per l'uso nei pazienti affetti da Niemann-Pick Disease Type C (NPC). MIPLYFFA™ è approvato per essere utilizzato in combinazione con miglustat per trattare le manifestazioni neurologiche della NPC in pazienti di età pari o superiore a due anni.
XOMA Royalty ha diritto a ricevere un royalty a cifra singola media sulle vendite di MIPLYFFA™ e fino a 52,6 milioni di dollari in traguardi. Questa approvazione segna MIPLYFFA™ come il sesto asset commerciale nel portafoglio di XOMA Royalty. Nel giugno 2023, XOMA Royalty ha acquisito questi diritti da LadRx per un pagamento iniziale di 5 milioni di dollari più una percentuale dei futuri traguardi legati a eventi.
XOMA Royalty anunció que Zevra Therapeutics ha recibido la aprobación de la FDA para MIPLYFFA™ (arimoclomol), la primera terapia aprobada para su uso en pacientes con la enfermedad de Niemann-Pick tipo C (NPC). MIPLYFFA™ está aprobado para ser utilizado en combinación con miglustat para tratar las manifestaciones neurológicas de la NPC en pacientes de dos años o más.
XOMA Royalty tiene derecho a recibir un royalty de un solo dígito medio sobre las ventas de MIPLYFFA™ y hasta 52,6 millones de dólares en hitos. Esta aprobación marca a MIPLYFFA™ como el sexto activo comercial en el portafolio de XOMA Royalty. En junio de 2023, XOMA Royalty adquirió estos derechos de LadRx por un pago inicial de 5 millones de dólares más una parte de los hitos futuros basados en eventos.
XOMA 로열티는 Zevra Therapeutics가 MIPLYFFA™ (아리모클로몰)에 대해 FDA 승인을 받았다고 발표했습니다. 이는 Niemann-Pick Disease Type C (NPC) 환자에게 사용이 승인된 최초의 치료제입니다. MIPLYFFA™는 NPC의 신경학적 증상을 치료하기 위해 miglustat와 함께 사용하도록 승인되었습니다. 이는 두 살 이상의 환자에서 사용할 수 있습니다.
XOMA 로열티는 MIPLYFFA™ 판매에 대한 중간 단일 디지털 로열티를 받을 권리가 있으며 5,260만 달러의 이정표에 도달할 수 있습니다. 이 승인은 MIPLYFFA™가 XOMA 로열티 포트폴리오의 여섯 번째 상업 자산으로 자리 매김합니다. 2023년 6월, XOMA 로열티는 LadRx로부터 500만 달러의 선불 지급과 함께 향후 이벤트 기반 이정표의 일부를 받기로 하였습니다.
XOMA Royalty a annoncé que Zevra Therapeutics a reçu l'approbation de la FDA pour MIPLYFFA™ (arimoclomol), la première thérapie approuvée pour une utilisation chez les patients atteints de la maladie de Niemann-Pick de type C (NPC). MIPLYFFA™ est approuvé pour une utilisation en association avec le miglustat afin de traiter les manifestations neurologiques de la NPC chez les patients âgés de deux ans et plus.
XOMA Royalty a le droit de recevoir un royalties à un chiffre moyen sur les ventes de MIPLYFFA™ et jusqu'à 52,6 millions de dollars en jalons. Cette approbation fait de MIPLYFFA™ le sixième actif commercial du portefeuille de XOMA Royalty. En juin 2023, XOMA Royalty a acquis ces droits auprès de LadRx pour un paiement initial de 5 millions de dollars plus une part des jalons futurs basés sur des événements.
XOMA Royalty gab bekannt, dass Zevra Therapeutics die FDA-Zulassung für MIPLYFFA™ (Arimoclomol) erhalten hat, die erste Therapie, die für die Anwendung bei Patienten mit Niemann-Pick-Krankheit Typ C (NPC) genehmigt wurde. MIPLYFFA™ ist in Kombination mit Miglustat zur Behandlung neurologischer Manifestationen von NPC bei Patienten ab zwei Jahren zugelassen.
XOMA Royalty hat Anspruch auf eine mittlere einstellige Lizenzgebühr auf die Verkaufszahlen von MIPLYFFA™ und bis zu 52,6 Millionen US-Dollar in Meilensteinen. Diese Genehmigung markiert MIPLYFFA™ als das sechste kommerzielle Asset im Portfolio von XOMA Royalty. Im Juni 2023 erwarb XOMA Royalty diese Rechte von LadRx für eine Vorauszahlung von 5 Millionen US-Dollar plus einen Anteil an zukünftigen ereignisbasierten Meilensteinen.
- FDA approval of MIPLYFFA™, the first therapy for Niemann-Pick Disease Type C
- XOMA Royalty to receive mid-single digit royalty on MIPLYFFA™ sales
- Potential for up to $52.6 million in milestone payments
- Addition of sixth commercial asset to XOMA Royalty's portfolio
- None.
MIPLYFFA™ is the first therapy approved for use in patients with NPC, a rare genetic disorder
XOMA Royalty is entitled to receive a mid-single digit royalty on MIPLYFFA™ sales and up to
MIPLYFFA™ is now the sixth commercial asset in XOMA Royalty’s portfolio
EMERYVILLE, Calif., Sept. 23, 2024 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today Zevra Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) for MIPLYFFA™ (arimoclomol). MIPLYFFA™ is approved for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients two years of age and older. It is the sixth commercial asset in XOMA Royalty’s growing royalty and milestone portfolio.
“With the approval of MIPLYFFA™, NPC patients in the United States now have access to the first FDA approved therapeutic in this rare, progressive and fatal neurodegenerative disease,” stated Owen Hughes, Chief Executive Officer of XOMA Royalty. “Based on the clinical data to date, we believe MIPLYFFA™ plus miglustat has the potential to improve outcomes and slow disease progression for many NPC patients.”
In June 2023, XOMA Royalty announced it had paid LadRx a
About XOMA Royalty Corporation
XOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial therapeutic candidates and commercial assets that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio with more than 70 assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit www.xoma.com or follow XOMA Royalty Corporation on LinkedIn.
Forward-Looking Statements/Explanatory Notes
Certain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding the amount of potential milestone and commercial payments to XOMA Royalty and other developments related to MIPLYFFA™ (arimoclomol), and the potential of XOMA Royalty’s portfolio of partnered programs and licensed technologies generating substantial milestone and royalty proceeds over time. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will,” “would,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA Royalty’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, and our third-party licensees will not be able to market them. Other potential risks to XOMA Royalty meeting these expectations are described in more detail in XOMA Royalty's most recent filing on Form 10-Q and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA Royalty's prospects. Any forward-looking statement in this press release represents XOMA Royalty's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA Royalty disclaims any obligation to update any forward-looking statement, except as required by applicable law.
EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development.
As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel
| Investor Contact | Media Contact |
| Juliane Snowden | Kathy Vincent |
| XOMA Royalty Corporation | KV Consulting & Management |
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| juliane.snowden@xoma.com | kathy@kathyvincent.com |
FAQ
What is MIPLYFFA™ and what has it been approved for?
How does the MIPLYFFA™ approval affect XOMA Royalty (XOMA)?
When did XOMA Royalty (XOMA) acquire the rights to MIPLYFFA™ royalties?
What is the significance of MIPLYFFA™'s approval for Niemann-Pick Disease Type C patients?
