Orphan Drug Designation Status for Acerta Pharma Drug Vindicates AstraZeneca PLC’s $4 Billion Deal

Orphan Drug Status for Acerta Drug Vindicates AstraZeneca’s $4 Billion Deal
February 25, 2016
By Alex Keown, BioSpace.com Breaking News Staff

LONDON – AstraZeneca 's acquisition of 55 percent of privately-held Acerta Pharma BV in December is showing signs of bolstering the deal after the European Medicines Agency recommended orphan drug status to acalabrutinib, Acerta’s key experimental drug, AstraZeneca announced Wednesday.

Acalabrutinib was recommended orphan drug status for the treatment of chronic lymphocytic leukemia (CLL) / small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL) and lymphoplasmacytic lymphoma (Waldenström’s macroglobulinaemia, MG), AstraZeneca said. Orphan Drug Designation is a status assigned to a medicine intended for use in rare diseases. To receive orphan drug designation in Europe, a drug must be intended for the “treatment, prevention or diagnosis of a disease that is life threatening” and has a commonality of up to five in 10,000 in the European Union.

Sean Bohen, AstraZeneca’s chief medical officer, said the EMA’s decision to grant acalabrutinib orphan status for three designations reinforces the company’s “strategic rationale” in its acquisition of controlling interest in Acerta. In a statement, Bohen said the company has a clear path “in developing a potential best-in-class medicine that could transform treatment for patients across a range of blood cancers.”

Prior to the deal with AstraZeneca, Netherlands-based Acerta published data for a Phase I and Phase II study, Acerta said its investigational drug acalabrutinib, showed a 95 percent response rate in patients with relapsed CLL. Acalabrutinib is a second-generation, selective and potent inhibitor of Bruton’s tyrosine kinase (Btk).

In addition to ongoing Phase II/III trials in CLL, MCL and WM, acalabrutinib is currently being tested in Phase I/II trials in monotherapy as well as in combination with immunotherapy or chemotherapies in a range of other blood cancers and solid tumors. CLL is a slow-growing blood and bone marrow cancer that accounts for approximately one in four cases of leukemia. MCL is an aggressive non-Hodgkin’s lymphoma (NHL) that is often fatal. WM is a rare, slow-growing cancer predominantly affecting older individuals that is typically fatal within five to nearly eleven years AstraZeneca said.

Based on its clinical trial data, AstraZeneca is likely banking on the possibility of acalabrutinib becoming a blockbuster treatment for CLL, looking to compete with Venetoclax, a CLL treatment being jointly developed by AbbVie and Genentech . During a panel at the American Society of Hematology meeting in Orlando, Fla. AbbVie announced Venetoclax has shown a nearly 80 percent positive response rate. Venetoclax is an investigational oral B-cell lymphoma-2 (BCL-2) inhibitor being investigated for the treatment of CLL and other blood disorders. The drug works by blocking the BCL-2 protein and “telling” the cancer cells to die, giving the healthy cells room to grow.

If acalabrutinib is approved in both the United States and the European Union, AstraZeneca will have the option to acquire the remaining 45 percent of Acerta for approximately $3 billion, Reuters reported.

A big money-making drug is something AstraZeneca is looking for to meet its revenue goal of $45 billion in sales by 2023. Acquiring new drugs through mergers and acquisitions will help the company offset declining revenues of drugs that are facing patent loss and increased generic competition, including the antacid Nexium and its heart drug Crestor. In December, AstraZeneca saw the approval of Tagrisso, its new treatment for non-small lung cancer.

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