Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe

Inês Martins, PhD avatar

by Inês Martins, PhD |

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EB-101 phase 3 trial

Krystal Biotech recently announced that the European Medicines Agency (EMA) has granted the company’s investigational therapy KB103 orphan medicinal product designation for the treatment of dystrophic epidermolysis bullosa (DEB).

KB103 is the first investigational non-infectious viral vector (HSV-1) gene therapy for DEB to receive this designation in Europe. In November 2017, the U.S. Food and Drug Administration also granted KB103 orphan drug status for the same indication.

DEB is a rare and incurable form of epidermolysis bullosa. The condition is characterized by blistering of the skin due to a lack of type 7 collagen (COL7) caused by mutations in the COL7A1 gene. COL7 keeps the epidermis (the skin’s upper layer) attached to the dermis (its lower layer). The lack of COL7 causes the epidermis to separate from the dermis, leading to blisters.

KB103 delivers a healthy and functional copy of the human COL7A1 gene directly to the patient’s skin cells to produce normal COL7 protein using a modified virus as a carrier (the HSV-1 virus). The goal is to prevent blistering.

Currently, no approved treatments for the underlying cause of DEB exist. KB103 could play an important role in this area of high unmet medical need.

In Europe, orphan medicinal product designation is available to products designed to treat life-threatening or chronic conditions that have a prevalence of no more than five in 10,000 European Union citizens. The designation is granted when no approved satisfactory treatment for the specific condition exists, and it offers a company market exclusivity for 10 years, along with protocol assistance from the EMA and access to tax and financial incentives.

“Being granted Orphan Medicinal Product Designation in Europe represents another important global milestone for our KB103 program,” Suma Krishnan, Krystal’s founder and chief operating officer, said in a press release. “Last month we filed an Investigational New Drug application for KB103 in the U.S. and intend to advance the clinical development of KB103 in U.S. in 2018 and in E.U. in 2019.”

In November 2017, Krystal Biotech also received clearance from the Recombinant DNA Advisory Committee for KB103. The federal advisory committee reviews research practices in basic and clinical recombinant DNA research or gene therapy.

Based on FDA and committee clearances, Krystal submitted an investigational new drug application with the FDA last March to start a Phase 1/2 clinical trial of KB103 as a treatment for DEB.

Approval of the application would enable the company to assess KB103’s safety, tolerability, and effectiveness in clinical trials.

In January 2018, the U.S. Patent and Trademark Office issued a patent covering Krystal’s HSV vectors and related gene therapies for the treatment of wounds and skin diseases.