The company’s KB105 treatment was previously approved by the US Food and Drug Administration in August 2018.

Krystal Biotech (NASDAQ:KRYS) has announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has given the company a positive opinion on its application for orphan designation of KB105 to treat transglutaminase-1 (TGM1) deficient autosomal recessive congenital ichthyosis (ARCI)

As quoted in the press release:

KB105 was previously granted orphan drug designation by the United States Food and Drug Administration in August 2018.

“We are delighted with the EMA COMP’s adoption of a positive opinion for KB105 orphan drug designation, which represents another important milestone for this clinical program,” said Suma Krishnan, chief operating officer of Krystal Biotech. “We believe KB105 has the potential to be the first gene therapy to bring hope to ARCI patients and their families and we look forward to announcing interim clinical results on two adult patients, currently enrolled in our Phase 1/2 clinical trial in 1H 2020.”

The positive opinion issued by COMP will be sent to the European Commission which is expected to grant the orphan designation within 30 days. Orphan designation in the EU allows Krystal Biotech to benefit from a number of key incentives, including reduced regulatory fees, protocol assistance, and market exclusivity, to develop a medicine for the treatment of a rare disease affecting not more than five in 10,000 people in the European Union.

TGM1-deficient ARCI is a debilitating rare skin disease characterized by excessive, thick scaling of the skin,­­­­ causing multiple chronic health conditions. There are approximately 23,000 cases of TGM1-deficient ARCI worldwide and about 400 new cases per year globally. Krystal’s approach is to use a non-replicating, non-integrating engineered HSV-1 vector to deliver the TGM1 gene to keratinocyte skin cells, potentially allowing them to produce the TGM1 protein that is lacking in this patient population. KB105 is designed to be an off-the-shelf treatment for TGM1-deficient ARCI that can be applied topically, directly to a patient’s skin.

Click here to read the full press release.

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