Recent Regulatory Submissions


Supplemental applications have been submitted for the cancer therapies Nubeqa and Tibsovo, the gout treatment Krystexxa, and Oxlumo, which treats a rare disease.

Bayer submits supplemental application for anti-cancer therapy Nubeqa.

Bayer has submitted a supplemental new drug application FDA, as well as the submission of a Variation Type II application to the European Medicines Agency for the oral androgen receptor inhibitor Nubeqa (darolutamide). Bayer is seeking approval for the use of Nubeqa in combination with docetaxel in patients with metastatic hormone-sensitive prostate cancer.

The submissions are supported by positive results from the phase 3 ARASENS trial, showing a statistically significant improvement in overall survival for Nubeqa plus androgen deprivation therapy and docetaxel in men with mHSPC. These results were presented in February at the 2022 ASCO GU Cancers Symposium and simultaneously published in The New England Journal of Medicine.

FDA grants priority review for Servier’s leukemia application.

Servier has submitted a supplemental new drug application (sNDA) for Tibsovo (ivosidenib) as a potential treatment for patients with previously untreated IDH1-mutated acute myeloid leukemia (AML). The sNDA was granted priority review, which accelerates the review and shortens the review time goal from 10 months to six months.

The application is based on the results of the AGILE study, a global, phase 3 trial in patients with previously untreated IDH1-mutated AML. Presented at the 2021 American Society of Hematology Annual Meeting, the data demonstrated that treatment with Tibsovo in combination with azacitidine improved event-free survival. In addition, the combination of showed a statistically significant improvement in overall survival, with a median overall survival of 24 months.

Tibsovo is currently approved in the United States as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory acute myeloid leukemia, and for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old. The therapy was also recently approved as a first and only targeted therapy for patients with previously treated IDH1-mutated cholangiocarcinoma.

Horizon submits supplemental BLA for Krystexxa.

The FDA has granted priority review for Horizon Therapeutics’ supplemental biologics license application (sBLA) to expand the label for Krystexxa (pegloticase) plus methotrexate, an immunomodulator commonly prescribed by rheumatologists. The Prescription Drug User Fee Act (PDUFA) action date is July 7, 2022.

Krystexxa is used to treat adult patients with uncontrolled chronic gout. Some patients, however, treated develop anti-drug antibodies, which can limit the effectiveness of treatment. Emerging data has suggested that adding methotrexate to the treatment can help to prevent these anti-drug antibodies, helping more patients achieve a durable response to therapy.

The application is based on the MIRROR randomized controlled trial, which showed 71.0% of patients (71 of 100) who were randomized to receive Krystexxa plus methotrexate achieved a complete serum uric acid response at least 80%. The trial demonstrated a 32.5-percentage point improvement compared with those randomized to Krystexxa with placebo (38.5%; 20 of 52 patients). No new safety concerns were identified.

Alnylan submits supplemental NDA for Oxlumo.

Alnylam Pharmaceuticals has submitted a supplemental new drug application (sNDA) for Oxlumo (lumasiran), an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase – for the reduction of plasma oxalate in the treatment of patients with advanced primary hyperoxaluria type 1 (PH1). The FDA has set a PDUFA date of October 6, 2022.

PH1 is a rare disease that affects the and results from the buildup of oxalate. It can result in kidney and bladder stones. PH1 is associated with a progressive decline in kidney function

The filing is based on the results of the ILLUMINATE-C study showing that Oxlumo can reduce plasma oxalate levels in patients with compromised renal function due to PH1, including those on hemodialysis. The study was a single arm phase 3 trial evaluating patients of all ages with several renal impairment.

In 2020, the FDA approved Oxlumo for the treatment of both adult and pediatric patients with PH1 and lower urinary oxalate levels. The EMA has also improved Oxlumo for the treatment of PH1 in all age groups.

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