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FDA Approves Increased Shelf Life and In-Use Storage at Room Temperature for Orfadin

WALTHAM, Mass.–(BUSINESS WIRE)–Sobi, a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families, announced today that the FDA approved a supplemental new drug application (sNDA) for Orfadin® (nitisinone) capsules in the U.S. The sNDA will allow for an extension of shelf life for the 20 mg capsules from 24 months to 36 months under refrigerated storage (2-8°C), and will also permit in-use storage at room temperature (25°C or less) for all strengths of Orfadin® capsules (2 mg, 5 mg, 10 mg and 20mg) for up to 45 days.

Orfadin® is approved in the U.S., Europe, and Canada for the treatment of hereditary tyrosinemia type-1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver, brain and kidney complications and can be fatal if untreated.

“The approval for increased shelf life and extended out-of-refrigeration time may help make Orfadin an even more flexible treatment option,” says Jon Miller, President of the Network of Tyrosinemia Advocates (NOTA). “Continued treatment improvements like this may help to lessen the burden of HT-1 for patients and their families.”

This sNDA approval for Orfadin® marks the latest milestone in a series of recent, positive updates around the treatment for patients with HT-1. In 2016, Sobi announced the approval of Orfadin® Oral Suspension (4 mg/mL) and the approval of the 20 mg dosing option in the U.S., allowing physicians to further personalize treatment.

“This approval is another important advancement for the HT-1 community. With every new treatment innovation, we have the opportunity to better the lives of people with rare disease,” says Rami Levin, President of Sobi in North America.

Twenty years ago, before pharmacological treatment was available, fewer than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday.i Today, treatment with Orfadin®, combined with dietary restriction of tyrosine and phenylalanine and more widespread newborn screening leading to early diagnosis, has greatly improved outcomes for HT-1 patients.ii

About Orfadin®

People with hereditary tyrosinemia type-1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver, renal and neurological complications. In the most common form of the disease, symptoms arise within the first six months of the child’s life. Approximately 1,000 persons worldwide are identified as living with HT-1 today.

Orfadin® (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine by-products in the body. Patients must maintain a special diet in combination with Orfadin® treatment as tyrosine is not adequately broken down. Orfadin®is a proprietary product and is developed by and marketed globally by Sobi. For more information about Orfadin®, visit www.orfadin.com.

Indication and Usage

Orfadin® is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).

Important Safety Information

  • Inadequate restriction of tyrosine and phenylalanine intake can result in elevations in plasma tyrosine.
  • Do not adjust Orfadin® dosage in order to lower the plasma tyrosine concentration.
  • Leucopenia and thrombocytopenia have been observed with treatment with Orfadin®. Monitor platelet and white blood cell counts regularly during Orfadin® therapy.
  • Most common adverse reactions (incidence >2%) are hepatic neoplasm, liver failure, thrombocytopenia, leucopenia, visual system complaints including conjunctivitis, corneal opacity, keratitis, and photophobia.
  • Use caution when administering Orfadin® with drugs that are metabolized by CYP2C9 because of a potential for increased systemic exposure of these drugs.
  • Orfadin® should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Based on animal data, Orfadin® may cause fetal harm.
  • Exercise caution when administering to a nursing woman. Because of the potential for serious adverse reactions in nursing infants from Orfadin®, a decision should be made to discontinue nursing or to discontinue the drug taking into account the importance of the drug to the mother.

For full prescribing information, please visit www.orfadin.com.

About Sobi™

Sobi is an international specialty healthcare company dedicated to rare diseases. Sobi’s mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. Sobi also markets a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies.

Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2015, Sobi had total revenues of SEK 5.2 billion (USD 608 M) and about 760 employees. The share (STO: SOBI) is listed on Nasdaq Stockholm. More information is available at www.sobi.com.

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