This section highlights FDA-related milestones and regulatory updates for drugs developed by Krystal Biotech (KRYS).
Over the past two years, Krystal Biotech has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
KB301, KB408, KB707, KB801, KB803, and VYJUVEK. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
KB301 - FDA Regulatory Timeline and Events
KB301 is a drug developed by Krystal Biotech for the following indication: Aesthetic skin conditions.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- KB301
- Announced Date:
- August 28, 2024
- Indication:
- Aesthetic skin conditions
Announcement
Krystal Biotech, Inc announced positive interim safety and efficacy results from both Cohorts 3 and 4 of PEARL-1, a Phase 1 study evaluating KB301, an investigational aesthetic treatment designed to deliver the COL3A1 transgene and increase type III collagen ("COL3") levels in the skin, for the improvement of lateral canthal lines at rest in Cohort 3 and for the improvement of dynamic wrinkles of the décolleté in Cohort 4.
AI Summary
Krystal Biotech, Inc. announced positive interim safety and efficacy results from Cohorts 3 and 4 of the PEARL-1 Phase 1 study evaluating KB301, an investigational aesthetic treatment. KB301 is designed to deliver the COL3A1 transgene to boost type III collagen levels in the skin. In Cohort 3, the treatment aimed to improve lateral canthal lines at rest, while Cohort 4 focused on reducing dynamic wrinkles of the décolleté. Both investigators and study subjects reported meaningful improvements in wrinkle appearance and other key skin attributes. The safety profile of KB301 was consistent with previous data, with all adverse events being mild-to-moderate and primarily related to the injection process. These encouraging results highlight KB301’s potential to offer a new approach for skin rejuvenation that goes beyond traditional toxins and fillers in addressing signs of aging.
Read Announcement
KB408 - FDA Regulatory Timeline and Events
KB408 is a drug developed by Krystal Biotech for the following indication: For the Treatment of Alpha-1 Antitrypsin Deficiency.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- KB408
- Announced Date:
- December 12, 2024
- Indication:
- For the Treatment of Alpha-1 Antitrypsin Deficiency
Announcement
Krystal Biotech, Inc announced today clinical data updates for both KB408 and KB407, the Company's clinical-stage, inhaled genetic medicine programs in Phase 1 for the treatment of rare respiratory diseases.
AI Summary
Krystal Biotech, Inc. announced new clinical data for its Phase 1 inhaled genetic medicine programs KB408 and KB407, developed to treat rare respiratory diseases. For KB408, designed for alpha-1 antitrypsin deficiency (AATD), early findings show clear SERPINA1 gene delivery and significant AAT expression in lung airway cells following a single dose. Some patients experienced an increase in AAT levels in the lungs and serum, with a corresponding decrease in unbound neutrophil elastase, highlighting the drug’s potential to provide effective, non-invasive therapy. KB407, targeted for cystic fibrosis (CF), has received conditional sanctioning from the Cystic Fibrosis Foundation, with initial dosing demonstrating a safe and tolerable profile. Both programs reported only mild to moderate, short-lived adverse events. These encouraging results support the further development and future clinical updates of Krystal Biotech’s innovative inhaled genetic medicine platform aimed at addressing unmet respiratory health needs.
Read Announcement
KB707 - FDA Regulatory Timeline and Events
KB707 is a drug developed by Krystal Biotech for the following indication: for the treatment of locally advanced or metastatic solid tumor malignancies.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- KB707
- Announced Date:
- April 22, 2024
- Indication:
- for the treatment of locally advanced or metastatic solid tumor malignancies.
Announcement
Krystal Biotech, Inc announced that the first patient was dosed in its Phase 1 clinical trial (KYANITE-1) evaluating inhaled KB707, a modified HSV-1 vector designed to deliver genes encoding both human interleukin-12 (IL-12) and interleukin-2 (IL-2) to the lung, for the treatment of patients with locally advanced or metastatic solid tumors of the lung.
AI Summary
Krystal Biotech, Inc. has dosed the first patient in its Phase 1 KYANITE-1 clinical trial. This study is testing inhaled KB707, a modified HSV-1 vector designed to deliver genes for both human interleukin-12 (IL-12) and interleukin-2 (IL-2) directly to the lungs. The treatment targets patients with locally advanced or metastatic solid tumors in the lung. The new inhaled approach could offer a safer and more effective way to deliver cytokine therapy, which has shown promise in triggering anti-tumor immune responses but has been limited by delivery challenges. Researchers hope that using the HSV-1-based gene delivery platform will overcome these issues and improve treatment outcomes for patients with difficult-to-treat lung cancers. The KYANITE-1 trial is open-label and will include dose escalation and expansion phases to assess the safety and potential benefits of KB707.
Read Announcement
KB801 - FDA Regulatory Timeline and Events
KB801 is a drug developed by Krystal Biotech for the following indication: For the Treatment of Neurotrophic Keratitis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- KB801
- Announced Date:
- July 9, 2025
- Indication:
- For the Treatment of Neurotrophic Keratitis
Announcement
Krystal Biotech, Inc announced that the first patient has been dosed in its Phase 1/2 clinical trial ("EMERALD-1"), a 2:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for the treatment of neurotrophic keratitis (NK).
AI Summary
Krystal Biotech, Inc. announced that the first patient has been dosed in its Phase 1/2 clinical trial, EMERALD-1, for the treatment of neurotrophic keratitis (NK). This study is a 2:1 randomized, double-masked, multicenter, placebo-controlled trial that will involve up to 27 adults with Stage 2 or Stage 3 NK. Participants are being treated with KB801, a redosable eye drop gene therapy designed to drive sustained local production of nerve growth factor (NGF) in the front of the eye. This innovative approach may help heal corneal epithelial defects associated with NK while reducing the frequent dosing burden seen with conventional therapies.
The primary goal of the trial is to assess the safety and tolerability of KB801, while the secondary objective is to evaluate its effectiveness in healing damaged corneal tissue over an 8-week period.
Read Announcement
KB803 - FDA Regulatory Timeline and Events
KB803 is a drug developed by Krystal Biotech for the following indication: In Patients with Dystrophic Epidermolysis Bullosa.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- KB803
- Announced Date:
- June 24, 2025
- Indication:
- In Patients with Dystrophic Epidermolysis Bullosa
Announcement
Krystal Biotech, Inc announced that that the first patient has been dosed in its Phase 3 clinical trial ("IOLITE"), an intra-patient, double-blind, multicenter, placebo-controlled study with crossover design evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients.
AI Summary
Krystal Biotech, Inc. announced that the first patient has been dosed in its Phase 3 clinical trial, IOLITE, which evaluates KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients. The IOLITE study features an intra-patient, double-blind, placebo-controlled, multicenter design with a crossover setup, meaning each patient receives both the treatment and a placebo at different times during the trial. KB803 is a redosable gene therapy delivered as an eye drop that aims to provide the eye’s epithelial cells with two copies of the COL7A1 transgene, encouraging local production of type VII collagen. This design could help restore full eye function and reduce vision loss from recurring abrasions. Patients aged six months and older will participate following a 12-week run-in period, and the trial will be conducted in a decentralized manner with treatment administered at home by a healthcare provider.
Read Announcement
VYJUVEK - FDA Regulatory Timeline and Events
VYJUVEK is a drug developed by Krystal Biotech for the following indication: Designed to treat DEB at the molecular level by providing the patient's skin cells the template to make normal COL7 protein.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- VYJUVEK
- Announced Date:
- April 28, 2025
- Indication:
- Designed to treat DEB at the molecular level by providing the patient's skin cells the template to make normal COL7 protein,
Announcement
Krystal Biotech, Inc announced that that on April 23, 2025, the European Commission (EC) granted marketing authorization to VYJUVEK® (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth.
AI Summary
Krystal Biotech recently announced that on April 23, 2025, the European Commission granted marketing authorization to VYJUVEK® (beremagene geperpavec-svdt) for treating wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the COL7A1 gene, starting from birth. This gene therapy works by delivering functional copies of the COL7A1 gene to address the root genetic cause of DEB, promoting proper collagen production and wound healing with the option for redosing. The approval allows for flexible dosing, so patients can receive treatment at home or in a healthcare setting, and administration by a patient or caregiver may be allowed under professional guidance. VYJUVEK’s approval marks an important milestone as the first corrective medicine approved in Europe for DEB, and it will be marketed in all EU member states as well as Iceland, Norway, and Liechtenstein.
Read Announcement- Drug:
- VYJUVEK
- Announced Date:
- February 28, 2025
- Indication:
- Designed to treat DEB at the molecular level by providing the patient's skin cells the template to make normal COL7 protein,
Announcement
Krystal Biotech, Inc. today welcomed the European Medicines Agency's (EMA's) announcement that its Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for the European Commission (EC) to approve VYJUVEK® (beremagene geperpavec-svdt, or B-VEC) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth.
AI Summary
Krystal Biotech announced exciting news as the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive recommendation. The CHMP advised that the European Commission approve VYJUVEK® (beremagene geperpavec-svdt, or B-VEC) for treating wounds in patients with dystrophic epidermolysis bullosa (DEB) who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene. Notably, the treatment is designed to be used from birth. The recommendation supports flexible administration, allowing the therapy to be applied in a clinic or at home, provided a healthcare professional appropriately instructs patients or caregivers. The final decision from the EC is expected in the second quarter of 2025 and will impact all European Union member states as well as Iceland, Norway, and Liechtenstein. This approval could mark a significant step forward in gene therapy for DEB, offering a simple and non-invasive approach to managing the condition.
Read Announcement
