Amgen and its subsidiary Onyx Pharmaceuticals, Inc announced that the European Medicines Agency (EMA) has accepted the Marketing Authorisation Application (MAA) of Kyprolis (carfilzomib) for Injection for the treatment of patients with relapsed multiple myeloma who have received at least one prior therapy. The MAA has been granted accelerated assessment by the EMA.

Kyprolis is a proteasome inhibitor, one of the classes of drugs used to treat multiple myeloma, an incurable blood cancer affecting approximately 89,000 people in Europe.1 Nearly all patients with the disease experience periods of remission, followed by relapses and eventually their disease becomes resistant to treatment.

"Achieving deep and durable responses for patients with relapsed multiple myeloma is critical towards extending the time they live without their disease progressing," says Pablo J. Cagnoni, MD, president, Onyx Pharmaceuticals, Inc. "We look forward to working with European regulators to potentially make this important medication available."

The MAA includes data from the phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial as well as other relevant data.

Kyprolis previously received orphan drug designation by the EMA in the European Union (EU). Orphan designation is granted for medicines intended for the treatment, prevention or diagnosis of a disease that is life threatening and has a prevalence in the EU of no more than five in 10,000. The intended medicine must aim to provide significant benefit to those affected by the condition.

Kyprolis was granted accelerated approval by the US Food and Drug Administration (FDA) in July 2012. Kyprolis is also approved for use in Argentina, Israel and Mexico.

Multiple myeloma is the second most common hematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow. Worldwide, nearly 230,000 people are living with multiple myeloma. In 2012, approximately 114,000 new cases were diagnosed and 80,000 people died. In Europe, approximately 89,000 people are living with multiple myeloma. Approximately 39,000 new cases were diagnosed and 24,000 people died in 2012. In the US, approximately 83,000 people were living with multiple myeloma in 2011. The estimated number of new cases in 2014 was 24,000 and the estimated number of deaths was 11,000.

The international, randomised phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial evaluated Kyprolis in combination with lenalidomide and low-dose dexamethasone, versus lenalidomide and low-dose dexamethasone alone, in patients with relapsed multiple myeloma following treatment with one to three prior regimens. The primary endpoint of the trial was progression-free survival, defined as the time from treatment initiation to disease progression or death. Secondary endpoints included overall survival, overall response rate, duration of response, disease control rate, health-related quality of life and safety. Patients were randomized to receive Kyprolis (20 mg/m2 on days 1 and 2 of cycle 1 only, escalating to 27 mg/m2 on days 8, 9, 15 and 16 of cycle 1 and continuing on days 1, 2, 8, 9, 15 and 16 of subsequent cycles), in addition to a standard dosing schedule of lenalidomide (25 mg per day for 21 days on, 7 days off) and low-dose dexamethasone (40 mg per week in 4 week cycles), versus lenalidomide and low-dose dexamethasone alone. The study randomized 792 patients at sites in North America, Europe and Israel.

The ASPIRE data were presented at the 56th Annual Meeting of the American Society of Hematology in December 2014 and published in the New England Journal of Medicine.

Onyx Pharmaceuticals received Scientific Advice from the EMA on the design and planned analysis of the ASPIRE trial and it was conducted under a Special Protocol Assessment (SPA) from the FDA.

On July 20, 2012, the US FDA granted accelerated approval of Kyprolis (carfilzomib) for Injection for the treatment of patients with multiple myeloma who have received at least two prior therapies including bortezomib and an immunomodulatory agent (IMiD) and have demonstrated disease progression on or within 60 days of completion of the last therapy. Approval was based on response rate. Clinical benefit, such as improvement in survival or symptoms, has not been verified.

Kyprolis is a product of Onyx Pharmaceuticals, Inc. Onyx Pharmaceuticals is a subsidiary of Amgen and holds development and commercialisation rights to Kyprolis globally, excluding Japan.