Acalabrutinib Secures Three Orphan Drug Designations in Europe, Validating AstraZeneca's $4 Billion Acquisition of Acerta Pharma

March 8, 2016 /Bioon/ -- Last December, AstraZeneca spent a hefty $4 billion to acquire a 55% stake in the private pharmaceutical company Acerta Pharma BV, a move that now appears very wise as its key drug, acalabrutinib, has been granted orphan drug status by the European Medicines Agency, with estimated peak sales reaching $5 billion post-launch!

Acalabrutinib has achieved successive successes in Europe, securing orphan drug designations for three indications: chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL), and lymphoplasmacytic lymphoma. Orphan drugs are designated for the treatment of rare diseases; in Europe, a rare disease is defined as one affecting fewer than 5 in 10,000 people.

Sean Bohen, President of AstraZeneca Pharmaceuticals, stated that the European Medicines Agency’s granting of three orphan drug indications for acalabrutinib fully reflects the “long-term strategic” vision behind the company’s acquisition of Acerta. The company has a clear strategy in treating various hematologic cancers, namely, to develop best-in-class medicines.

Acalabrutinib is a second-generation Bruton's tyrosine kinase (BTK) inhibitor. This drug exerts its effect by irreversibly binding to BTK, which is a component of a specific protein complex that transmits signals from the surface of chronic lymphocytic leukemia (CLL) cells to genes in the nucleus, thereby promoting cancer cell survival and proliferation. By inhibiting BTK, acalabrutinib blocks these growth signals in CLL cells, ultimately leading to cancer cell death.

The early clinical data for acalabrutinib are also encouraging. Results from a Phase I/II clinical trial, recently published in The New England Journal of Medicine, showed a response rate of 95% in patients with chronic lymphocytic leukemia (CLL), the most common form of leukemia in adults.

Its favorable clinical efficacy positions it as a potential blockbuster drug for the treatment of chronic lymphocytic leukemia (CLL), posing strong competition to venetoclax, which was jointly developed by AbbVie and Roche. This agent is an investigational B-cell lymphoma-2 (BCL-2) inhibitor. BCL-2 is a protein that prevents apoptosis in certain cells, including lymphocytes, and is highly expressed in cancer cells arising in lymph nodes, the spleen, and other organs of the immune system. Venetoclax is designed to selectively inhibit the function of BCL-2, thereby restoring cellular signaling pathways and inducing cancer cell self-destruction, ultimately achieving antitumor effects.

If Acerta Pharma’s anticancer drug receives approval from regulatory authorities in the United States and Europe, AstraZeneca may exercise its option to acquire the remaining 45% equity stake in Acerta Pharma for $3 billion.

To achieve its 2023 annual sales target of $45 billion, AstraZeneca is seeking blockbuster drugs. Acquiring new medications through mergers and acquisitions can help the company navigate the patent cliff and the quagmire of generic competition. AstraZeneca faces patent expirations for its flagship gastric drug esomeprazole (Nexium) and its lipid-lowering drug rosuvastatin calcium (Crestor). Meanwhile, the company’s non-small cell lung cancer treatment, Tagrisso, was approved for market launch last December, and Acalabrutinib is poised to become a powerful addition to strengthen its new drug pipeline. (Bioon.com)

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Original Source:Orphan Drug Designation Status for Acerta PharmaDrug Vindicates AstraZeneca PLC (AZN)’s $4 Billion Deal