This section highlights FDA-related milestones and regulatory updates for drugs developed by Ligand Pharmaceuticals (LGND).
Over the past two years, Ligand Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
QTORIN, ZELSUVMI, Lasix®, and FCX-007. 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.
QTORIN FDA Regulatory Timeline and Events
QTORIN is a drug developed by Ligand Pharmaceuticals for the following indication: For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
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:
- QTORIN
- Announced Date:
- May 4, 2026
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, Inc. announced that the first patients have been dosed in LOTU, a multicenter Phase 2 clinical trial designed to evaluate the safety and efficacy of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of clinically significant angiokeratomas.
AI Summary
Palvella Therapeutics, Inc. announced that the first patients have been dosed in LOTU, a multicenter Phase 2 clinical trial testing QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for clinically significant angiokeratomas. The study will evaluate the safety and effectiveness of the topical rapamycin formulation as a potential treatment for this rare, chronic and often debilitating lymphatic malformation. There are currently no FDA‑approved therapies for angiokeratomas.
The Phase 2 LOTU trial is a single‑arm, baseline‑controlled study expected to enroll up to 15 subjects at leading vascular anomaly centers and high‑volume dermatology centers across the U.S. Palvella plans to report topline results in the second half of 2027. An estimated more than 50,000 patients in the U.S. have been diagnosed with angiokeratomas, highlighting the unmet medical need this trial aims to address.
Read Announcement- Drug:
- QTORIN
- Announced Date:
- March 27, 2026
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, Inc., announced two poster presentations at the 2026 American Academy of Dermatology Annual Meeting to be held March 27-31 in Denver, Colorado.
AI Summary
Palvella Therapeutics announced two poster presentations at the 2026 American Academy of Dermatology Annual Meeting, March 27–31 in Denver. Poster #76954 will present QTORIN™ rapamycin, a 3.9% anhydrous gel formulation designed to improve dermal bioavailability by addressing rapamycin’s crystallization, stability, and skin-penetration challenges. Poster #76929 reports a qualitative patient and caregiver interview study describing the burden of porokeratosis, a rare genetic skin disease marked by numerous pre-cancerous, itchy lesions and major functional and psychosocial impacts.
Palvella says it is advancing QTORIN™ rapamycin across multiple serious, rare skin diseases and vascular malformations that currently lack FDA-approved therapies, including microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas, with a fourth target planned to be announced in the second half of 2026. QTORIN™ rapamycin and QTORIN™ pitavastatin are investigational only and have not been approved by the FDA or other regulatory agencies.
Read Announcement- Drug:
- QTORIN
- Announced Date:
- February 24, 2026
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, Inc announced positive topline results from the Company's Phase 3 SELVA study of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs).
AI Summary
Palvella Therapeutics announced positive topline results from the Phase 3 SELVA study of QTORIN™ 3.9% rapamycin anhydrous gel for microcystic lymphatic malformations. The primary endpoint was met with a statistically significant mean improvement of +2.13 on the Microcystic Lymphatic Malformation Investigator Global Assessment (mLM-IGA) (p<0.001). The pre-specified key secondary endpoint and all four secondary efficacy endpoints also reached statistical significance (all p<0.001). Among participants aged ≥6 who completed the efficacy period, 95% improved on the mLM-IGA at Week 24 and 86% were rated "Much Improved" (+2) or "Very Much Improved" (+3). A blinded photo-based clinician scale (mLM-MCSS) improved by a mean of 3.36 points (p<0.001).
QTORIN™ rapamycin was well tolerated with no drug-related serious adverse events reported and systemic rapamycin levels below 2 ng/mL at all timepoints. Ninety-eight percent of participants who completed the efficacy period elected to continue in the treatment extension. Palvella plans to submit a New Drug Application in the second half of 2026, with potential U.S. approval in the first half of 2027; if approved, QTORIN™ could become the first approved therapy for the estimated more than 30,000 people with microcystic LMs in the U.S.
Read Announcement- Drug:
- QTORIN
- Announced Date:
- February 23, 2026
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, Inc. announced that it will host a conference call and webcast tomorrow, Tuesday, February 24, 2026, at 8:00am ET to discuss topline results from the Phase 3 SELVA clinical trial assessing the efficacy and safety of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations.
AI Summary
Palvella Therapeutics announced it will host a conference call and webcast tomorrow, Tuesday, February 24, 2026, at 8:00 a.m. ET to discuss topline results from the Phase 3 SELVA clinical trial. The SELVA study evaluated the efficacy and safety of QTORIN™ 3.9% rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations. During the call, company leaders plan to present key outcome measures, initial interpretations of the data, and how the topline findings address the trial’s goals.
The discussion will focus on both efficacy signals and safety observations and what those results could mean for patients and next steps in development. Palvella may outline timing for full data release, further analyses, and potential regulatory or clinical plans. QTORIN™ rapamycin is investigational and has not been approved by the FDA or other regulatory authorities. Investors, clinicians, patient advocates, and media are invited to join the live webcast or conference call for the company’s summary and commentary.
Read Announcement- Drug:
- QTORIN
- Announced Date:
- November 5, 2025
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, announced a new product candidate, QTORIN™ pitavastatin, for the treatment of disseminated superficial actinic porokeratosis (DSAP).
AI Summary
Disseminated superficial actinic porokeratosis, or DSAP, is a serious skin disease with growing, premalignant lesions. It affects over 50,000 people in the U.S. and causes chronic loss of skin integrity that can harm quality of life. No approved treatments exist.
Palvella Therapeutics has announced QTORIN™ pitavastatin, a new topical medicine designed to target the root cause of DSAP. It works by blocking the mevalonate pathway in skin cells, where genetic mutations cause harmful intermediate buildup. Published case studies on off-label statin creams support this approach. With higher potency than other inhibitors, it may offer a next-generation option.
Palvella plans to start a Phase 2 trial of QTORIN™ pitavastatin in the second half of 2026. The company will discuss this candidate in a webcast conference call today, November 5, 2025, at 8:30 a.m. ET.
Read Announcement- Drug:
- QTORIN
- Announced Date:
- April 2, 2025
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, Inc announced a late-breaking oral presentation at the upcoming 15th World Congress of Pediatric Dermatology, taking place April 8-11, 2025, in Buenos Aires, Argentina..
AI Summary
Palvella Therapeutics, Inc. announced a late-breaking oral presentation at the upcoming 15th World Congress of Pediatric Dermatology in Buenos Aires, Argentina, from April 8-11, 2025. The company will share findings from the SELVA Phase 3 clinical trial, which evaluates QTORIN™ rapamycin 3.9% anhydrous gel for the treatment of microcystic lymphatic malformations in patients aged 3 years and older. The study, being presented by Dr. Amy Paller, focuses on a fit-for-purpose primary endpoint that measures the gel’s effectiveness against this rare condition, characterized by malformed lymphatic vessels that cause persistent leakage, bleeding, and related complications. This innovative approach is particularly important because there are currently no approved treatments for this lifelong, debilitating genetic disease. The presentation is expected to provide crucial insights into a potential new therapeutic option for affected patients.
Read Announcement- Drug:
- QTORIN
- Announced Date:
- October 3, 2024
- Indication:
- For the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).
Announcement
Palvella Therapeutics, Inc announced the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has awarded the company a grant of up to $2.6 million to support the ongoing Phase 3 SELVA trial of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs).
Read Announcement
ZELSUVMI FDA Regulatory Events
ZELSUVMI is a drug developed by Ligand Pharmaceuticals for the following indication: For the Treatment of Molluscum Contagiosum.
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:
- ZELSUVMI
- Announced Date:
- December 4, 2025
- Indication:
- For the Treatment of Molluscum Contagiosum
Announcement
Pelthos Therapeutics Inc. announced it has signed its first commercial agreement to expand patient access for ZELSUVMI™ (berdazimer) topical gel, 10.3%, for the treatment of molluscum contagiosum in adults and pediatric patients one year of age and older ("ZELSUVMI").
AI Summary
Pelthos Therapeutics announced its first commercial agreement to expand patient access for ZELSUVMI (berdazimer) topical gel, 10.3%, for treating molluscum contagiosum in adults and children one year and older. The company partnered with a Group Purchasing Organization that works with one of the largest pharmacy benefit managers in the U.S., which manages prescription benefits for more than 20 million covered lives. Formulary inclusion updates for ZELSUVMI began December 1, 2025, a step Pelthos says will broaden coverage and distribution networks and make the treatment easier to obtain for patients and caregivers.
Pelthos’ leadership emphasized that the PBM partnership supports their patient services plan and efforts to reduce access barriers. ZELSUVMI, launched in July 2025, is a once-daily topical nitric oxide–releasing gel intended for convenient at-home use, including on sensitive areas, providing an additional treatment option for people affected by this contagious skin condition.
Read Announcement- Drug:
- ZELSUVMI
- Announced Date:
- July 10, 2025
- Indication:
- For the Treatment of Molluscum Contagiosum
Announcement
Pelthos Therapeutics Inc. announced the launch of ZELSUVMI™ (berdazimer) topical gel, 10.3%, for the treatment of molluscum contagiosum (molluscum) in adults and pediatric patients one year of age and older.i,ii
AI Summary
Pelthos Therapeutics Inc. has launched ZELSUVMI™ (berdazimer) topical gel, 10.3% for treating molluscum contagiosum in adults and pediatric patients aged one year and older. This prescription medication offers a once-daily, at-home option that allows patients, parents, and caregivers to manage the condition without frequent office visits. Molluscum contagiosum is a highly contagious viral skin infection that affects millions each year, making this new treatment a welcome option for many.
In clinical trials, ZELSUVMI showed effective results, with nearly one-third of patients achieving complete clearance of lesions. The product is now available at retail pharmacies, ASPN pharmacy services, and through mail-order pharmacies, aiming to ease the burden of this common pediatric and adult skin condition and provide a convenient treatment option outside of traditional clinical settings.
Read Announcement
Lasix® ONYU FDA Regulatory Events
Lasix® ONYU is a drug developed by Ligand Pharmaceuticals for the following indication: Home Treatment for Edema in Heart Failure Patients.
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:
- Lasix® ONYU
- Announced Date:
- October 9, 2025
- Indication:
- Home Treatment for Edema in Heart Failure Patients
Announcement
Ligand Pharmaceuticals nnounced that its partner SQ Innovation Inc. has received approval from the U.S. Food and Drug Administration (FDA) for Lasix® ONYU (furosemide injection), a novel drug-device combination for the treatment of edema (due to fluid overload) in adult patients with chronic heart failure.
AI Summary
Ligand Pharmaceuticals’ partner SQ Innovation Inc. has won FDA approval for Lasix® ONYU, a new drug-device combo to treat edema from fluid overload in adults with chronic heart failure. Lasix ONYU delivers a high-concentration furosemide injection (80 mg/2.67 mL) under the skin, so selected patients can receive treatment at home without a nurse or doctor present. The system uses a small, reusable Infusor unit good for 48 doses and a single-use sterile cartridge.
This product is the 17th to use Ligand’s Captisol® technology, which improves drug solubility, stability, and bioavailability. Lasix ONYU aims to match hospital IV diuresis while lowering costs and reducing the need for hospital stays. SQ Innovation expects to launch Lasix ONYU in the fourth quarter of 2025, offering a more convenient, cost-effective option for heart failure patients, healthcare providers, and payors.
Read Announcement
FCX-007 FDA Regulatory Events
FCX-007 is a drug developed by Ligand Pharmaceuticals for the following indication: In patients with dystrophic epidermolysis bullosa (DEB).
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:
- FCX-007
- Announced Date:
- February 25, 2025
- Indication:
- In patients with dystrophic epidermolysis bullosa (DEB).
Announcement
Ligand Pharmaceuticals announced it has closed a royalty financing agreement with Castle Creek Biosciences, Inc. to support the Phase 3 clinical study of D-Fi (FCX-007), Castle Creek's lead candidate, in patients with dystrophic epidermolysis bullosa (DEB).
AI Summary
Ligand Pharmaceuticals recently closed a royalty financing agreement with Castle Creek Biosciences to support the Phase 3 clinical trial of D-Fi (FCX-007), the company’s leading candidate for treating dystrophic epidermolysis bullosa (DEB). Under this agreement, Ligand invested $50 million and led a syndicate of co-investors who contributed an additional $25 million. In return, they will receive a high-single-digit royalty on the worldwide sales of D-Fi.
Dystrophic epidermolysis bullosa is a severe, rare genetic skin disorder, and D-Fi is an innovative gene therapy that uses a patient’s own modified cells. Both companies expressed confidence that this funding will drive the trial’s progress and help bring a much-needed treatment closer to patients. This strategic collaboration underlines Ligand’s commitment to backing promising therapies while expanding its portfolio of revenue-generating assets.
Read Announcement
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