Dystrophic Epidermolysis Bullosa (DEB) Treatment Granted OMPD in Europe

This morning, Krystal Biotech, Inc announced that its product, KB103, was granted Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency (EMA) for the treatment of dystrophic epidermolysis bullosa (DEB).

KB103 is a replication-defective, non-integrating viral vector that has been engineered to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is the replication-deficient, non-integrating viral vector that is able to penetrate skin cells more efficiently than other viral vectors. Due to its high payload capacity, it is able it to accommodate large or multiple genes. In addition, its low immunogenicity makes it an appropriate choice for direct and repeated delivery to the skin.

DEB is painful and often fatal skin blistering condition caused by a lack of collagen protein in the skin. These “butterfly children” (as they are called) experience painful skin blisters due to a mutation in the gene COL7A1. KB103 uses a modified herpes simplex (HSV-1) virus as a vehicle in an attempt to deliver the missing or mutated COL7A1 gene to the patients.²

Suma Krishnan, founder and chief operating officer of Krystal, shared her excitement with the drug’s progress. “Being granted Orphan Medicinal Product Designation in Europe represents another important global milestone for our KB103 program. Last month, we filed an Investigational New Drug application for KB103 in the US and intend to advance the clinical development of KB103 in US in 2018 and in EU in 2019."¹

OMPD is a designation in Europe that is only granted when there are no approved or satisfactory treatments for a specific condition. OMPD pertains to products that aim at treating life-threatening or chronically debilitating conditions that have patient prevalence in the European Union (“EU”) of no more than five in 10,000 people.

Stateside, the US Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to KB103 in November 2017. In an exclusive interview with Krish Krishnan, CEO of Krystal Biotech, Rare Disease Report learned of the drug’s progress for FDA approval in the States.

In the interview, Krish Krishnan echoed a similar, positive projection regarding KB103’s timeline in the US. “We have a lot of work to do; the clinical data has to pan out. We are hopeful to file for approval on KB103 in 2020 and launch thereafter. However, should KB103 prove to be efficacious in the upcoming Phase 1/2 clinical trial, Krystal intends to work with the Agency and patient advocacy groups to pursue KB103 for compassionate or expanded use.”

In an exclusive quote to Rare Disease Report, Krish Krishnan further emphasized the drug's strides in Europe. “The Orphan Designation in Europe is an important regulatory milestone as we further our development of KB103 in dystrophic epidermolysis bullosa where there are no treatment options presently. We received an Orphan Drug Designation from the FDA last year and recently filed an IND application on KB103 in the United States. We look forward to commencing clinical trials in the US shortly and in the EU in 2019.”

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References:

1. Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe for Dystrophic Epidermolysis Bullosa. http://globenewswire.com/news-release/2018/04/19/1481647/0/en/Krystal-Biotech-s-KB103-Receives-Orphan-Medicinal-Product-Designation-in-Europe-for-Dystrophic-Epidermolysis-Bullosa.html?ev=1). Accessed April 19, 2018.
2. Is New Treatment on the Horizon for Dystrophic Epidermolysis Bullosa? https://www.mdmag.com/medical-news/is-new-treatment-on-the-horizon-for-deb. Accessed April 19, 2018.