Top 3 new drugs to treat rare diseases

FDA recently approved a new treatment for 2 rare types of non-Hodgkin lymphomas, as well as a new drug to treat hereditary angiodema and 1 for Fabry disease.

Here are the top 3 new drugs to treat rare diseases.

• Mogamulizumab-kpkc (Oteligeo, Kyowa Kirin) injection for intravenous use treats adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS), rare, hard-to-treat types of non-Hodgkin lymphoma.

Oteligeo is the first FDA approval of a drug specifically for SS, and is a new treatment option for patients with MF.

Related: FDA okays radiation drug for ultra-rare cancers

The approval “fills an unmet medical need for these patients,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a statement.

Oteligeo’s approval was based on a clinical trial of 372 patients with relapsed MF or SS who received either Poteligeo or a type of chemotherapy called vorinostat. Progression-free survival (the amount of time a patient stays alive without the cancer growing) was longer for patients taking Poteligeo (median of 7.6 months) compared to patients taking vorinostat (median 3.1 months).

  2.  Lanadelumab-flyo (Takhzyro, Shire Plc) was approved for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older. HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of edema (swelling) in various parts of the body.

Takhzyro is the only monoclonal antibody (mAb) that provides targeted inhibition of plasma kallikrein, an enzyme which is chronically uncontrolled in people with HAE, to help prevent attacks, according to a statement from Shire.

“HAE attacks are painful, debilitating, and potentially life threatening. Takhzyro provides the HAE community with a new option for the prevention of HAE attacks,” said Anthony J. Castaldo, president of the U.S. Hereditary Angioedema Association, in the statement.

Takhzyro has a half-life of around 2 weeks and is administered as 1 subcutaneous self-injection every 2 weeks at the recommended starting dose.

Related: Major PBM’s formulary exclusions cause concerns

  3.  Migalastat (Galafold, Amicus Therapeutics US) is the first oral medication to treat adults with Fabry disease, a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs.

The drug is indicated for adults with Fabry disease who have a genetic mutation determined to be responsive to treatment with Galafold based on laboratory data.

“Thus far, treatment of Fabry disease has involved replacing the missing enzyme that causes the particular type of fat buildup in this disease. Galafold differs from enzyme replacement in that it increases the activity of the body’s deficient enzyme,” said Julie Beitz, MD, director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research, in a statement from FDA.

The efficacy of Galafold was demonstrated in a six-month, placebo-controlled clinical trial in 45 adults with Fabry disease. Patients treated with Galafold over 6 months had a greater reduction in globotriaosylceramide (GL-3) in blood vessels of the kidneys (as measured in kidney biopsy samples) as compared to patients on placebo.The safety of Galafold was studied in 4 clinical trials which included a total of 139 patients with Fabry disease.

Read more: Novel gene-silencing drug holds promise for this rare disease