Hybrigenics gets Orphan Drug designation for the treatment of Acute Myeloid Leukemia

LeukemiaHybrigenics (ALHYG), a French bio-pharmaceutical company with a focus on research and development of new treatments against proliferative diseases, has announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to inecalcitol for the treatment of acute myeloid leukemia (AML) in the United States. AML is designated as an orphan disease in the United States, Europe and Japan.

The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. At the time of the enactment of the Orphan Drug Act, the United States Congress made several factual findings.  Congress observed that there are many diseases and conditions, such as Huntington’s disease, ALS (Lou Gehrig’s disease), Tourette syndrome, and muscular dystrophy, for example, which affect such small numbers of individuals that the diseases and conditions are considered rare in the United States.  As a result, adequate drugs have not been developed and realistically cannot be expected to be developed because companies would likely incur a financial loss in the pursuit of such drugs, given the high cost of developing drugs.

At the time the Orphan Drug Act passed, Congress made the determination that a seven (7) year period of exclusivity was appropriate if a company produced a drug to treat a rare disease that affects less than 200,000 people in the United States.  Orphan designation is also possible if a disease affects more than 200,000 people if it can also be shown that there is no reasonable expectation that the cost of developing the drug could be recouped absent this period of exclusivity. This “patent-like” exclusivity is available even if the drug is not novel and non-obvious.

In addition to providing a seven-year term of market exclusivity upon final FDA approval, orphan drug designation allows the company receiving the designation to leverage a wide range of financial and regulatory benefits, including government and institutional grants, and waiver of FDA user fees for the potential submission of a New Drug Application.


Acute myeloid leukemia

Acute myeloid leukemia is a type of cancer that affects the blood and bone marrow. AML is characterized by a fast-increasing overproduction of immature white blood cells, called myeloblasts. These cells rapidly crowd the bone marrow, soon preventing it from making normal blood cells. They can also spill out into the blood stream and circulate around the body.  Inadequate numbers of red cells and platelets being made by the marrow cause anemia, and easy bleeding and/or bruising.

World-wide, acute myeloid leukemia (AML) is the second most frequent form of leukemia, behind only chronic lymphocytic leukemia, and accounts for about 30% of all leukemic patients. According to the Leukemia & Lymphoma Society, in 2014 there were 53,380 people diagnosed with leukemia in the United States. The most frequent form of leukemia in the United States is AML, with 18,900 new cases diagnosed during 2014, which represents 36% of all newly diagnosed leukemia cases in the United States.

This favorable decision by the FDA is based on in vitro and in vivo preclinical evidence showing the synergy between inecalcitol and azacytidine or decitabine, two hypo-methylating anticancer drugs, to inhibit the growth of human AML cell lines in vitro and, in vivo, to prolong the survival of mice in two different experimental models of AML. The molecular basis of their synergy with inecalcitol, a vitamin D receptor agonist is the result of unmasking the gene coding for vitamin D receptors (by reducing the methylation of its promoter region). As a consequence, more vitamin D receptors are expressed and available to be activated by inecalcitol, resulting in an improved efficacy to limit leukemia progression over the hypo-methylating agents alone.

Inecalcitol alone has already been studied in a Phase II clinical trial in chronic lymphocytic leukemia, with an inhibitory effect has been shown in half of the treated patients. Another Phase II clinical study has recently been launched in combination with imatinib (Gleevec®, Novartis) in chronic myeloid leukemia. Enrollment is ongoing in the imatinib clinical study.

AML can occur at any age but is more common in adults over the age of 60 years. Treatment needs to begin soon after AML is diagnosed, as it progresses very quickly. Chemotherapy is the main form of treatment for AML; occasionally, a stem cell transplant may be used. Despite available treatments, AML shows the lowest 5-year survival rate of all leukemias: 25 per cent in the US and 19 per cent in Europe.


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