Amgen receives FDA Breakthrough Therapy Designation for investigational BiTE® antibody blinatumomab in acute lymphoblastic leukemia

AmgenAmgen (NASDAQ:AMGN) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to investigational bispecific T cell engager (BiTE®) antibody blinatumomab, for adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL), a rapidly progressing cancer of the blood and bone marrow(1).

The Breakthrough Therapy Designation was based on the results of a Phase 2 trial of 189 adult patients with Ph- relapsed/refractory B-precursor ALL treated with blinatumomab. Data from the Phase 2 trial were most recently presented at the 50th Annual Meeting of the American Society of Clinical Oncology (ASCO) and the 19th Congress of the European Hematology Association (EHA).

"There is a high unmet need for new medicines to treat relapsed and refractory ALL patients, who have very few treatment options," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "The results from the Phase 2 trial evaluating blinatumomab in adult patients with relapsed or refractory ALL are encouraging and provide a strong basis for a regulatory filing later this year and potential approval in this serious disease."

The FDA states that Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy Designation conveys all of the fast-track program features, more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review(2).

In the U.S. alone, it is estimated that over 6,000 cases of ALL were diagnosed in 2013, and in the European Union, more than 7,000 cases of ALL are diagnosed each year(3,4). In adult patients with relapsed or refractory ALL, median overall survival is just three to five months(5).

About BiTE® Technology
Bispecific T cell engager (BiTE®) antibodies are a type of immunotherapy being investigated for use in fighting cancer by helping to engage the body's immune system to detect and target malignant cells. The modified antibodies are designed to engage two different targets simultaneously, thereby juxtaposing T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. BiTE antibodies help place the T cells within reach of the targeted cell, with the intent of allowing it to inject toxins and trigger the cell to die (apoptosis). BiTE antibodies are currently being investigated for their potential to treat a wide variety of cancers.

About Blinatumomab
Blinatumomab is an investigational BiTE® antibody designed to direct the body's cell-destroying T cells against target cells expressing CD19, a protein found on the surface of B-cell derived leukemias and lymphomas. Blinatumomab is the first of the BiTE antibodies and Amgen has received orphan drug designation from the FDA for the treatment of ALL, chronic lymphocytic leukemia (CLL), hairy cell leukemia, prolymphocytic leukemia and indolent B cell lymphoma and from the European Medicines Agency for the treatment of indolent B cell lymphoma, ALL, CLL and mantle cell leukemia (MCL). Blinatumomab is also being investigated for its potential to treat pediatric relapsed/refractory ALL, relapsed/refractory Philadelphia positive (Ph+) B-precursor ALL, minimal residual disease positive (MRD+) B-precursor ALL, relapsed/refractory non-Hodgkin's lymphoma (NHL), including relapsed/refractory diffuse large B-cell lymphoma (DLBCL).

About ALL
Acute lymphoblastic leukemia (ALL) is an aggressive cancer of the blood and bone marrow - the spongy tissue inside bones where blood cells are made(1). The disease progresses rapidly and affects immature blood cells, rather than mature ones(1). Worldwide, ALL accounts for more than 12 percent of leukemia. Of the 42,000 people diagnosed worldwide, 31,000 will die from the disease. Patients with ALL have abnormal white blood cells (lymphocytes) that crowd out healthy white blood cells, red blood cells and platelets, leading to infection, anemia (fatigue), easy bleeding and serious side effects(6).

About Amgen
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its biologics manufacturing expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be the world's largest independent biotechnology company, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

1. Mayo Clinic. "Acute lymphocytic leukemia." Available at: http://www.mayoclinic.com/health/acute-lymphocytic-leukemia/DS00558. Accessed on May 28, 2014.
2. U.S. Food and Drug Administration. "Frequently Asked Questions: Breakthrough Therapies." Available at: http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/ucm341027.htm. Accessed on May 28, 2014.
3. Siegel R, Naishadham D, Jemal A. Cancer statistics, 2013. Ca Cancer J Clin. 2013;63:11-30.
4. Gatta G, Maarten van der Zwan J, Casali P, et. al. Rare cancers are not so rare: The rare cancer burden in Europe. Eur J Cancer. 2011;47:2493-2511.
5. Advani A.S. New immune strategies for the treatment of acute lymphoblastic leukemia: Antibodies and chimeric antigen receptors. Hematology Am Soc Hematol Educ Program. 2013;2013:131-7. Retrieved from: http://asheducationbook.hematologylibrary.org/content/2013/1/131.long.
6. Mayo Clinic. "Acute lymphocytic leukemia: symptoms". Available at: http://www.mayoclinic.com/health/acute-lymphocytic-leukemia/DS00558/DSECTION=symptoms. Accessed on May 28, 2014.

Most Popular Now

Primary endpoint met in COMET-TAIL Phase III trial…

GlaxoSmithKline plc (LSE/NYSE: GSK) and Vir Biotechnology, Inc. (Vir) (Nasdaq: VIR) announced headline data from the randomised, multi-centre, open-label COMET-TAIL Phase...

Merck and Ridgeback's molnupiravir, an oral COVID-…

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics announced that the United Kingdom Medicines and Healthcare products Re...

Johnson & Johnson COVID-19 vaccine named one o…

The editors of Time announced that the Johnson & Johnson COVID-19 vaccine has been selected as one of Time's Best Inventions of 2021. The vaccine, for which Johnson & ...

New target for COVID-19 vaccines identified

Next generation vaccines for COVID-19 should aim to induce an immune response against 'replication proteins', essential for the very earliest stages of the viral cycle, c...

Safety concerns raised for neuroblastoma candidate…

St. Jude Children's Research Hospital scientists looking for drugs to improve survival of children with high-risk neuroblastoma found a promising candidate in CX-5461. Th...

Two billion doses of AstraZeneca’s COVID-19 vaccin…

AstraZeneca and its partners have released for supply two billion doses of their COVID-19 vaccine to more than 170 countries across every continent on the planet in the l...

Pfizer's novel COVID-19 oral antiviral treatment c…

Pfizer Inc. (NYSE: PFE) today announced its investigational novel COVID-19 oral antiviral candidate,PAXLOVID™, significantly reduced hospitalization and death, based on a...

'Dancing molecules' successfully repair severe spi…

Northwestern University researchers have developed a new injectable therapy that harnesses “dancing molecules” to reverse paralysis and repair tissue after severe spinal ...

Repurposing a familiar drug for COVID-19

For the past year and a half, the COVID-19 pandemic has continued to engulf the globe, fueled in part by novel variants and the uneven distribution of vaccines. Every day...

Researchers reveal a strategy for next-generation …

A study led by the Garvan Institute of Medical Research has revealed a guide to developing COVID-19 vaccines that both prevent the coronavirus from infecting human cells ...

A target for potential cancer drugs may, in fact, …

In recent years, much scientific effort and funding has focused on developing drugs that target an enzyme with the unwieldy name of Src homology 2-containing protein tyro...

A commonly found parasite could treat certain type…

Scientists have discovered that a deadly parasite, known to cause ill health in pregnant women and immunocompromised patients, could potentially be used to treat various ...