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Horizon Therapeutics Wins US FDA Acceptance of New Drug Application to Make Procysbi Available as Oral Granules in Packets.

M2 PHARMA-July 22, 2019-Horizon Therapeutics Wins US FDA Acceptance of New Drug Application to Make Procysbi Available as Oral Granules in Packets


- The US Food and Drug Administration has accepted Irish drug developer Horizon Therapeutics plc's (NASDAQ: HZNP) New Drug Application for Procysbi (Cysteamine Bitartrate) Delayed-Release Oral Granules in Packets, the company said.

If approved by the FDA, this new dosage form would provide another option for patients, in addition to the currently available PROCYSBI delayed-release capsules.

The capsules are FDA-approved for children one year of age and older and adults living with nephropathic cystinosis.

The FDA is expected to make a decision on the approval of the proposed new dosage form of granules in packets in 2020.

The proposed new dosage form would provide patients and their caregivers with the option of opening a packet containing small, compact granules and sprinkling on certain foods and juice (or administering through a feeding tube).

If approved, these packets would provide an alternative to the currently available option of opening individual capsules to remove the granules.

Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.

If untreated, elevated cystine accumulation leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.

It is estimated that only about 2,000 people worldwide are currently diagnosed with nephropathic cystinosis.

Nephropathic or "classic infantile" cystinosis the most common and most severe form of the disease is typically diagnosed in infancy and requires lifelong cystine depleting therapy.

Procysbi (cysteamine bitartrate) delayed-release capsules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

Horizon is focused on researching, developing and commercialising medicines that address critical needs for people impacted by rare and rheumatic diseases.

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Publication:M2 Pharma
Date:Jul 22, 2019
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