The Prescription Drug User Fee Act (PDUFA) date refers to the deadline set by the US Food and Drug Administration (FDA) for reviewing a New Drug Application (NDA) or Biologics License Application (BLA) and making a final decision on marketing approval. The typical period for review is 10 months after the drug application has been accepted by the Agency. For drugs that have Priority Review, the review period is reduced to 6 months from the time of application acceptance.
Ensitrelvir for COVID-19 Postexposure Prophylaxis
PDUFA date: June 16, 2026
Ensitrelvir is an investigational oral antiviral under FDA review for the prevention of COVID-19 following exposure to an infected individual. It is designed to suppress the replication of SARS-CoV-2 by selectively inhibiting the main protease (3-CL).
The NDA submission includes data from the phase 3 SCORPIO-PEP trial (ClinicalTrials.gov Identifier: NCT05897541), which evaluated the effectiveness of ensitrelvir for postexposure prophylaxis of COVID-19. Study participants were randomly assigned to receive ensitrelvir or placebo once daily for 5 days. Treatment was initiated within 3 days of symptom onset in an infected household member.
Results demonstrated that ensitrelvir significantly reduced the risk of developing COVID-19 by day 10 compared with placebo. If approved, ensitrelvir would be the first oral antiviral therapy for preventing COVID-19 after exposure.
Cystisinicline for the Treatment of Nicotine Dependence
PDUFA date: June 20, 2026
The FDA is reviewing cytisinicline, a plant-derived alkaloid, for the treatment of nicotine dependence in adults. Cytisinicline as a partial agonist at the α4β2 nicotinic acetylcholine receptor. Interaction with these receptors reduces withdrawal symptoms, as well as the reward and satisfaction associated with smoking.
The NDA includes data from two phase 3 trials: ORCA-2 (ClinicalTrials.gov Identifier: NCT04576949) and ORCA-3 (ClinicalTrials.gov Identifier: NCT05206370). Findings showed treatment with oral cytisinicline significantly improved smoking abstinence rates compared with placebo. Moreover, nicotine cravings were significantly reduced in cytisinicline-treated patients, resulting in less nicotine intake. The most commonly reported adverse events in both trials were insomnia, abnormal dreams, headaches, and nausea.
If approved, cytisinicline would be the first new pharmacotherapy for smoking cessation since the approval of varenicline in 2006.
Nanoencapsulated Sirolimus Plus Pegadricase for Uncontrolled Gout
PDUFA date: June 27, 2026
Sobi is seeking approval of nanoencapsulated sirolimus plus pegadricase (NASP) for the treatment of uncontrolled gout. NASP is a sequential 2-component infusion therapy that consists of pegadricase, a pegylated uricase, and tolerogenic nanoencapsulated sirolimus, an mTOR inhibitor immunosuppressant that prevents the production of anti-drug antibodies. The novel therapy is designed to lower serum uric acid (sUA) levels in patients with uncontrolled gout, thereby reducing the potential for flares and joint deformity.
The BLA is supported by data from the DISSOLVE 1 (ClinicalTrials.gov Identifier: NCT04513366) and DISSOLVE 2 (ClinicalTrials.gov Identifier: NCT04596540) trials, both double-blind, placebo-controlled studies that evaluated the safety and efficacy of NASP in patients with gout refractory to conventional therapy. Findings showed both trials met the primary endpoint of achieving and maintaining reduction in sUA less than 6mg/dL for at least 80% of the time during month 6.
Rapid and sustained reductions in sUA were observed following the first treatment with NASP and throughout the study period. In the 6-month extension phase of the DISSOLVE I trial, 75% of patients who completed 6 months of NASP as a responder continued to respond to therapy at month 12.
Olezarsen for the Treatment of Severe Hypertriglyceridemia
PDUFA: June 30, 2026
The FDA granted Priority Review to the supplemental NDA for olezarsen in the treatment of severe hypertriglyceridemia. Olezarsen is a ligand-conjugated antisense oligonucleotide that inhibits the production of apolipoprotein C-III, resulting in increased clearance of triglyceride and very low-density lipoprotein. It is administered as a subcutaneous injection.
The sNDA is based on data from the phase 3 CORE (ClinicalTrials.gov Identifier: NCT05079919) and CORE2 (ClinicalTrials.gov Identifier: NCT05552326) studies, which included adult participants with triglyceride levels of 500mg/dL or greater. Findings showed olezarsen significantly reduced triglyceride levels and the risk of acute pancreatitis at 6 months compared with placebo.
Olezarsen is currently marketed under the brand name TryngolzaTM as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.
Veligrotug for the Treatment of Thyroid Eye Disease
PDUFA: June 30, 2026
Priority Review has been granted to veligrotug for the treatment of thyroid eye disease (TED). The humanized monoclonal antibody inhibits the activity of insulin-like growth factor-1 receptor (IGF-1R), a key driver of TED symptoms including proptosis and diplopia.
The BLA includes data from the phase 3 THRIVE (ClinicalTrials.gov Identifier: NCT05176639) and THRIVE-2 (ClinicalTrials.gov Identifier: NCT06021054) trials, which evaluated veligrotug in patients with active and chronic TED, respectively. Results showed both trials met the primary and secondary endpoints, demonstrating significant and rapid improvements in proptosis and diplopia with veligrotug compared with placebo.
The treatment is delivered as an intravenous infusion given every 3 weeks for a total of 12 weeks (5 infusions total).
This article originally appeared on MPR