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
KB707, KB407, KB801, VYJUVEK, KB304, KB803, and KB408. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
KB707 FDA Regulatory 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:
- February 9, 2026
- Indication:
- for the treatment of locally advanced or metastatic solid tumor malignancies.
Announcement
Krystal Biotech, Inc announced today that the United States Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, the Company's redosable immunotherapy designed to drive sustained, localized expression of interleukin-2 and interleukin-12 in the tumor microenvironment, for the treatment of advanced or metastatic non-small cell lung cancer (NSCLC).
AI Summary
Krystal Biotech announced that the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, the company’s redosable immunotherapy for advanced or metastatic non-small cell lung cancer (NSCLC). KB707 is designed to produce sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) directly in the tumor microenvironment, aiming to boost immune activity against tumors while limiting systemic side effects. The therapy is intended to be dosed repeatedly to maintain local cytokine levels and enhance anti-tumor responses.
The RMAT designation recognizes KB707 as a promising regenerative medicine therapy and can help speed development through more frequent FDA interactions, guidance on trial design, and potential eligibility for accelerated approval pathways. This could shorten the path to later-stage trials and, if successful, earlier patient access.Read Announcement
KB407 FDA Regulatory Events
KB407 is a drug developed by Krystal Biotech for the following indication: Cystic Fibrosis.
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:
- KB407
- Announced Date:
- January 8, 2026
- Indication:
- Cystic Fibrosis
Announcement
Krystal Biotech, Inc announced a positive interim clinical update from the highest dose cohort of CORAL-1, the Company's multi-center, dose escalation Phase 1 study evaluating KB407 in patients with cystic fibrosis (CF), confirming the successful lung delivery and expression of wild-type cystic fibrosis transmembrane conductance regulator (CFTR) protein following inhaled administration of KB407.
AI Summary
Krystal Biotech reported a positive interim update from the highest dose group of CORAL-1, a Phase 1 study of inhaled KB407 for cystic fibrosis. Molecular testing showed delivery and expression of full‑length, wild‑type CFTR protein in clinically relevant ciliated and secretory airway cells — including patients with class I mutations — a first for the field.
Seven patients received four daily inhaled doses (about 1 billion PFU each); bronchoscopies were successful in six patients and KB407 transduction was confirmed in all six. The portion of conducting airway cells showing CFTR or viral marker expression ranged from 29.4% to 42.1% across those patients. KB407 was generally well tolerated; most related adverse events were mild or moderate. One serious adverse event (asthma exacerbation) occurred after bronchoscopy, was deemed procedure‑related, and resolved in five days.
These results show broad lung delivery and reproducible transduction across genetic backgrounds and modulator status, supporting the company’s move toward a repeat‑dosing study to further evaluate KB407’s safety and efficacy.Read Announcement
- Drug:
- KB407
- Announced Date:
- January 7, 2026
- Indication:
- Cystic Fibrosis
Announcement
Krystal Biotech, Inc announced that it will host an investor conference call and webcast tomorrow, Thursday, January 8, 2026, at 4:30 pm ET, to disclose an interim clinical update from CORAL-1, the Company's multi-center, dose escalation Phase 1 study evaluating KB407 in patients with cystic fibrosis.
AI Summary
Krystal Biotech announced it will hold an investor conference call and webcast tomorrow, Thursday, January 8, 2026, at 4:30 pm ET to share an interim clinical update from CORAL-1, its multi-center, dose-escalation Phase 1 study of KB407 in people with cystic fibrosis. The update will emphasize results from the highest dose cohort and include molecular assessments showing KB407 transduction and expression of wild-type CFTR protein after inhaled dosing.
Investors and the public can join the live webcast at https://www.webcaster5.com/Webcast/Page/3018/53466. An archived recording will be posted in the Investors section of Krystal Biotech’s website and remain available for at least 30 days for anyone who cannot listen live.
Read Announcement
KB801 FDA Regulatory 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:
- October 14, 2025
- Indication:
- For the Treatment of Neurotrophic Keratitis
Announcement
Krystal Biotech, Inc. announced today that the United States Food and Drug Administration (FDA) granted platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1 (HSV-1) viral vector used in the Company's redosable eye drop gene therapy KB801, currently under evaluation in a randomized placebo controlled trial for the treatment of neurotrophic keratitis (NK).
Read Announcement- 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
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:
- September 15, 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 today that the United States Food and Drug Administration (FDA) approved a label update for VYJUVEK® (beremagene geperpavec-svdt) that expands the VYJUVEK eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and provides patients full flexibility with respect to VYJUVEK application and managing wound dressings.
AI Summary
Krystal Biotech, Inc. announced today that the FDA approved a label update for VYJUVEK (beremagene geperpavec-svdt) to treat wounds in patients with dystrophic epidermolysis bullosa (DEB) from birth. The update makes infants and newborns eligible for treatment, expanding access to the youngest patients.
The new label also gives full flexibility on how VYJUVEK can be used. Patients and caregivers may now apply the gel themselves at home, fitting treatment into daily routines. Wound dressings can be removed during the next dressing change, rather than waiting a full 24 hours, making it easier to integrate VYJUVEK into existing care.
Krystal’s CEO, Krish S. Krishnan, said these changes reinforce VYJUVEK as the most flexible and convenient DEB therapy in the United States. Brett Kopelan, Executive Director of debra of America, added that caregiver application and improved dressing management will boost convenience and quality of life for DEB patients.
Read Announcement- Drug:
- VYJUVEK
- Announced Date:
- July 25, 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 today that on July 24, 2025, Japan's Ministry of Health, Labour and Welfare (MHLW) granted marketing authorization to VYJUVEK® (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB), starting from birth.
AI Summary
On July 24, 2025, Japan’s Ministry of Health, Labour and Welfare granted marketing authorization to VYJUVEK® for treating wounds in patients with dystrophic epidermolysis bullosa (DEB) from birth. The therapy delivers functional COL7A1 gene copies to heal wounds and support type VII collagen production with repeat dosing. It is the first genetic medicine approved in Japan for DEB.
The MHLW approval allows dosing at home or in healthcare settings with patients or family members administering treatment. Genetic testing is not required. Home dosing was approved after safety and environmental reviews under Japan’s Cartagena Act. VYJUVEK’s safety profile and low environmental risk supported at-home use.
Krystal Biotech expects reimbursement procedures to finish and launch VYJUVEK in Japan by the end of 2025. The approval offers a long-awaited option for DEB patients, improving healing and quality of life.
Read Announcement- 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
KB304 FDA Regulatory Events
KB304 is a drug developed by Krystal Biotech for the following indication: for the treatment of wrinkles of the décolleté.
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:
- KB304
- Announced Date:
- July 24, 2025
- Indication:
- for the treatment of wrinkles of the décolleté.
Announcement
Jeune Aesthetics, Inc. ("Jeune"), a wholly owned subsidiary of Krystal Biotech, Inc. announced positive safety and efficacy results, including significant improvements in key skin aesthetic attributes such as wrinkles and elasticity, in PEARL-2, a randomized, double-blind, placebo-controlled Phase 1 study evaluating KB304, for the treatment of wrinkles of the décolleté.
AI Summary
On July 24, Jeune Aesthetics, a subsidiary of Krystal Biotech, announced positive safety and efficacy results from PEARL-2, a Phase 1, randomized, placebo-controlled trial of KB304 injections for décolleté wrinkles.
Nineteen subjects enrolled: 12 got KB304 and seven got placebo. One KB304 patient withdrew, leaving 18 evaluated over three months.
Investigators and patients saw significant gains on the Global Aesthetic Improvement Scale: at one to three months, all KB304 subjects improved by at least one point, compared to placebo. By month 3, 82% reported better wrinkle satisfaction versus placebo.
Additional improvements in elasticity, crepiness, hydration and radiance appeared in all treated subjects each month. Adverse events were mild to moderate, declined after the first dose, and no serious drug-related events occurred.
Jeune will meet the FDA to finalize Phase 2 design and expects to begin Phase 2 trials in early 2026.
Read Announcement
KB803 FDA Regulatory 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
KB408 FDA Regulatory 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
KB301 FDA Regulatory 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
