FDA approves a new dosing option of ORFADIN® (nitisinone) -- 20 mg Capsule

WALTHAM, Mass., June 15, 2016 /PRNewswire/ -- Swedish Orphan Biovitrum AB (Sobi), an international specialty healthcare company dedicated to rare diseases, announced today that the Food and Drug Administration (FDA) has approved ORFADIN® 20 mg Capsule for the treatment of Hereditary Tyrosinemia Type 1 (HT-1). This is the second time this year Sobi is expanding the ORFADIN dosing options for HT-1 patients. ORFADIN is indicated for use in combination with dietary restriction of tyrosine and phenylalanine.    

Treatment with ORFADIN is weight-based. As patients grow, they will need progressively higher doses. Having a higher strength capsule may help minimize the number of capsules needed to achieve treatment levels of adolescent and adult patients.

"We are excited to offer this new dosing option. By introducing ORFADIN 20 mg Capsule, older patients may be able to decrease the number of capsules they take every day," said Len Walt, Vice President & Head of Medical Affairs, North America. "Our hope is to better meet patients' needs as they progress through different life stages."

Earlier this year, Sobi announced the approval of Orfadin Oral Suspension.  With the availability of the 20 mg Capsule, physicians can further personalize treatment, whether they are infants or adults.  ORFADIN is available in capsules (2 mg, 5 mg, 10 mg, and 20 mg) and oral suspension (4 mg/mL).

HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver and kidney complications and can be fatal if untreated. Before pharmacological treatment was available, less than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday.¹ Treatment with ORFADIN combined with dietary restriction of tyrosine and phenylalanine plus more widespread newborn screening leading to early diagnosis has greatly improved outcomes for HT-1 patients.² 

ORFADIN 20 mg Capsules will be available in September 2016.

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. ORFADIN® (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic by-products in the body. In the most common form of the disease, symptoms arise within the first six months of the child's life. Patients must maintain a special diet in combination with ORFADIN treatment as tyrosine is not adequately broken down. Approximately 1,000 persons are identified as living with HT-1 today. 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. Our 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. We also market 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 3.2 billion (USD 385 M) and approximately 700 employees. The share (STO:SOBI) is listed on NASDAQ Stockholm. More information is available on www.sobi.com.

¹ van Spronsen FJ, Thomasse Y, Smit GP, et al. Hepatology. 1994;20(5):1187-1191
² Orfadin Prescribing Information Rev Jun 2016

CONTACT: Jennifer Paganelli, W2O Group, 347-658-8290, [email protected]

SOURCE Sobi

Related Links

http://www.sobi.com