US FDA beviljad prioriterad granskning för aktuell JAK-hämmare Opzelura Cream

Incyte recently announced that the U.S. Food and Drug Administration (FDA) has accepted Opzelura (ruxolitinib, 1.5% cream) supplementary new drug application (sNDA) and granted priority review: the drug is a non-steroidal, anti-inflammatory, Topical JAK inhibitors for the treatment of vitiligo (vitiligo) in adults and adolescents (age> 12 years). The FDA has designated the sNDA's "Prescription Drug User Charges Act (PDUFA)" target date as April 18, 2022. In October of this year, the European Medicines Agency (EMA) accepted Opzelura's marketing authorization application (MAA) and has initiated a formal review process: it is used for adults and adolescents (age>12 år) för att behandla icke-segmentell vitiligo med ansiktspåverkan .

If approved, ruxolitinib cream will be the first and only drug used to treat vitiligo for repigmentation. Vitiligo is a chronic autoimmune disease characterized by depigmentation of the skin, which is a skin disease caused by the loss of pigment{{0}}producing cells, melanocytes, which often affects beauty. Vitiligo affects about 0.5 percent -2.0 percent of the world's population. Currently, there is no drug therapy approved by the US FDA or EU EMA for the treatment of vitiligo. The disease can occur at any age, although many people with vitiligo will experience initial symptoms before the age of 20.

Ruxolitinib cream is Incyte's patented formulation of selective Janus kinase 1 and Janus kinase 2 (JAK1/JAK2) inhibitor ruxolitinib, designed for topical application. Incyte has global rights to develop and commercialize ruxolitinib cream. Currently, ruxolitinib cream is in phase 3 clinical development: (1) for the treatment of mild to moderate atopic dermatitis (TRuE-AD project); (2) for the treatment of vitiligo in adolescents and adults (TRuE-V project) .

I september 2021 godkände amerikanska FDA Opzelura (ruxolitinib-kräm) för kort- och icke-varaktiga kroniska behandlingar. Att få aktuella receptbelagda behandlingar misslyckades med att adekvat kontrollera sjukdomen eller när dessa behandlingar är oönskade och icke-immuna-komprometterade mild till måttlig atopisk dermatit (AD) ungdom (ålder över eller lika med 12 år) och vuxna patienter.

Det är värt att nämna att Opzelura är den första och enda aktuella JAK-hämmaren godkänd av US FDA. Studier har visat att oregleringen av JAK-STAT-vägen leder till huvuddragen i AD, såsom klåda, inflammation och dysfunktion av hudbarriären. I en klinisk fas 3-studie minskade behandlingen med Opzelura signifikant hudinflammation och klåda i samband med AD. Och att minska klåda kan potentiellt förbättra nyckelsjukdomsrelaterade-resultat och livskvalitet för AD-patienter.

The regulatory application of ruxolitinib cream for the treatment of vitiligo is based on the results of the key Phase 3 TRuE-V clinical trial project. The data showed that the two phase 3 clinical studies of the project reached the primary and key secondary endpoints: after 24 weeks of treatment, compared with the excipient cream treatment group, the facial and whole body skin lesions in the ruxolitinib cream treatment group were restored The color has improved significantly. In this project, no clinically significant site reactions were reported for ruxolitinib cream for 24 weeks, and the overall safety is good. 

Jonathan Dickinson, Executive Vice President and European General Manager of Incyte, said: "EMA's acceptance of ruxolitinib cream MAA marks an important milestone for the group of patients with vitiligo. For them, their daily lives are usually greatly affected, and treatment options are currently limited. . We are committed to listening to the opinions of the patient community to understand how we can help meet unmet needs and support healthcare providers to better manage this challenging disease. We look forward to working with regulatory agencies to improve This new potential therapy brings qualified patients."

Ruxolitinib is the active pharmaceutical ingredient of Incyte's oral drug Jakafi. The drug has been approved for 3 indications in the United States: (1) Treatment of adult patients with polycythemia (PV) who have an insufficient or intolerant response to sulfhydryluria; (2) Treatment of middle and high-risk adult patients with myelofibrosis (MF), including primary MF, post-PV MF, post-essential thrombocythemia MF; (3) treatment of steroid-refractory acute graft-versus-host disease (GVHD) patients. Among them, the third indication was approved by the FDA in May 2019, and it was the first drug approved for the treatment of this indication. Jakafi is sold by Incyte in the United States, and Novartis is sold under the Jakavi brand name in markets outside the United States.

För närvarande utvecklar Concert också en ruxolitinib-molekyl modifierad med deuteriumkemisk teknologi-CTP-543. I en klinisk fas II-studie har den visat stark effekt vid behandling av alopecia areata. Alopecia areata är en autoimmun sjukdom som orsakar helt eller delvis håravfall. Den deuteriumkemiska modifieringen av ruxolitinib kan förändra dess humana farmakokinetik och därigenom förbättra dess användning som behandling för alopecia areata. I USA har FDA beviljat CTP-543 fast track-status för behandling av alopecia areata.