Raptor Pharmaceuticals Corp.
) recently announced that Procysbi has been approved for
marketing as an orphan medicinal product in the EU for the
treatment of proven nephropathic cystinosis. Approval was
expected as the European Medicines Agency's (EMA) Committee for
Medicinal Products for Human Use (CHMP) had rendered a positive
opinion regarding Procysbi in Jun 2013.
The company had also received a positive opinion on Procysbi
from the European Medicines Agency's (EMA) Committee for Orphan
Medicinal Products (COMP) for orphan medicinal product
exclusivity in Jul 2013 for the treatment of proven nephropathic
The orphan drug status will provide Procysbi 10 years of
market exclusivity in the EU.
We remind investors that Procysbi was approved by the U.S.
Food and Drug Administration (FDA) on Apr 30, 2013 for the
treatment of nephropathic cystinosis in adults as well as
children above 6 years of age. The drug was launched in Jun 2013.
Moreover, Procysbi enjoys orphan drug status in the U.S.
On receiving U.S. approval for Procysbi, Raptor Pharma
received the second tranche of $25 million, under its loan
agreement with HealthCare Royalty Partners (HC Royalty). In Dec
2012, the companies had signed a loan agreement under which
Raptor Pharma agreed to borrow $50 million for Procysbi in two
tranches. The first tranche of $25 million was received in Dec
2012. The loan is expected to mature in 2019.
Meanwhile, Procysbi is being evaluated for other indications
as well. Procysbi is currently in a phase II/III study for the
treatment of Huntington's disease. The company has orphan drug
status in the U.S. for Procysbi for the Huntington's disease
indication. The company expects data from this study in the first
quarter of 2014.
Procysbi is being studied in a phase IIb trial for the
treatment of nonalcoholic fatty liver in children (NAFLD). Raptor
Pharma expects to complete patient enrollment in the first half
Raptor Pharma currently carries a Zacks Rank #3 (Hold).
Currently, companies which look attractive include
Gilead Sciences Inc.
) with a Zacks Rank #1 (Strong Buy), and
) with a Zacks Rank #2 (Buy).
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