The firm is on track to launch the gene therapy for dystrophic epidermolysis bullosa, called Vyjuvek in the US, in Germany in Q2 2025.

CHMP opinion now expected in 1Q 2025No Major Objections outstanding; continue to expect Germany launch in 2Q 2025PITTSBURGH, Dec. 09, 2024 ...

Investigators concluded that the encouraging oncologic outcomes coupled with a favorable safety profile warrant further ...

Krystal Biotech (KRYS) announced that the European Medicines Agency’s, or EMA’s, Committee for Medicinal Products for Human Use, or CHMP, ...

In severe cases, such as Recessive Dystrophic EB, bandage changes to protect wounds and prevent blister infection are required every day and can last three to four hours at a time. The two-year ...

The Food and Drug Administration (FDA) has accepted for review the resubmitted Biologics License Application (BLA) for prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis ...

A resubmission Biologics License Application for prademagene zamikeracel (pz-cel) as a potential treatment for recessive dystrophic epidermolysis bullosa (RDEB) has been accepted by the FDA, according ...

as a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.

The Food and Drug Administration (FDA) has accepted for review the resubmitted Biologics License Application (BLA) for prademagene zamikeracel for the treatment of recessive dystrophic ...

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CHMP opinion now expected in 1Q 2025 No Major Objections outstanding; continue to expect Germany launch in 2Q 2025 PITTSBURGH, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") ...

Neurons face unique challenges of transporting nascent autophagic vacuoles (AVs) from distal axons toward the soma, where mature lysosomes are mainly located. Autophagy defects have been linked to ...