Updates from FDA
APhA Staff
New drugs
TALETRECTINIB
(Ibtrozi—Nuvation Bio)
Drug class: Ibtrozi is a kinase inhibitor.
Indication: Ibtrozi is indicated for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer.
Recommended dosage and administration: The recommended dosage is 600 mg orally once daily on an empty stomach. Continue treatment until disease progression or unacceptable toxicity occurs.
Common adverse effects: The most frequently reported adverse reactions include diarrhea, nausea, vomiting, dizziness, rash, constipation, fatigue, increased ALT and AST, decreased neutrophils, and increased creatine phosphokinase.
Warnings and precautions: Monitor liver function tests prior to initiating, every 2 weeks during the first 2 months of treatment, then monthly thereafter as clinically indicated, with more frequent testing in patients who develop transaminase elevations. Based on severity and resolution, withhold and then resume at a reduced dose or permanently discontinue. Monitor patients for new or worsening pulmonary symptoms indicative of interstitial lung disease or pneumonitis. Immediately withhold Ibtrozi in patients with suspected interstitial lung disease or pneumonitis.
Based on severity and resolution, resume at the same or a reduced dose or permanently discontinue. Monitor ECG and electrolytes prior to initiating and periodically during treatment. Based on severity and resolution, withhold and then resume at the same or a reduced dose or permanently discontinue. Monitor serum uric acid levels prior to initiating and periodically during treatment. Initiate treatment with urate-lowering medications as clinically indicated. Withhold and resume at the same or a lower dose or permanently discontinue based on severity. Monitor serum creatine phosphokinase levels during treatment in patients reporting unexplained muscle pain, tenderness, or weakness. Based on severity, withhold and resume at same or reduced dose upon improvement. Promptly evaluate patients with signs or symptoms of fractures.
Ibtrozi can cause fetal harm. Advise patients of this potential risk to a fetus and to use effective contraception. Advise patients not to breastfeed during treatment. Avoid concomitant use with strong and moderate CYP3A inducers, strong and moderate CYP3A inhibitors, and drugs that prolong the QTc interval. Also avoid concomitant use with PPIs and H2 receptor antagonists. If an acid-reducing agent cannot be avoided, administer Ibtrozi 2 hours before or 2 hours after taking a locally acting antacid.
SUNVOZERTINIB
(Zegfrovy—Dizal Pharmaceutical Co)
Drug class: Ibtrozi is a kinase inhibitor.
Indication: Ibtrozi is indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.
Recommended dosage and administration: The recommended dosage is 200 mg orally once daily taken with food.
Common adverse effects: The most frequently reported adverse reactions include diarrhea, rash, decreased appetite, stomatitis, fatigue, nausea, paronychia, vomiting, constipation, musculoskeletal pain, pruritus, dry skin, UTI, abdominal pain, decreased weigh, decreased lymphocytes, increased lipase, decreased hemoglobin, increased amylase, increased creatine kinase, decreased neutrophils, decreased potassium, increased aspartate aminotransferase, increased alamine aminotransferase, decreased sodium, increased magnesium, and increased alkaline phosphatase.
Warnings and precautions: Monitor patients for new or worsening pulmonary symptoms indicative of interstitial lung disease or pneumonitis. Immediately withhold Zegfrovy in patients with suspected interstitial lung disease or pneumonitis and discontinue if diagnosis is confirmed. Administer Zegfrovy with food to reduce GI adverse reactions. Monitor patients for nausea, vomiting and diarrhea, and provide supportive care, including anti-diarrheals, anti-emetics, or fluid replacement, as indicated. Withhold, reduce the dose, or permanently discontinue Zegfrovy based on severity. Monitor patients for rash and dermatologic adverse reactions. Instruct patients to use alcohol-free emollient cream during treatment and to avoid the use of irritating skin products. Withhold, reduce the dose, or permanently discontinue Zegfrovy based on severity. Promptly refer patients presenting with eye symptoms suggestive of keratitis to an ophthalmologist. Advise discontinuation of contact lenses until ocular symptoms are evaluated. Zegfrovy can cause fetal harm. Advise patients of reproductive potential of the potential risk to a fetus and to use effective nonhormonal contraception. Advise patients not to breastfeed during treatment. Avoid concomitant use with strong CYP3A inhibitors and if concomitant use cannot be avoided, reduce the Zegfrovy dose. Avoid concomitant use with strong and moderate CYP3A inducers and if concomitant use cannot be avoided, increase the Zegfrovy dose. Monitor for increased adverse reactions during concomitant use with P-gp or BCRP substrates. Avoid concomitant use with hormonal contraceptives.
SEBETRALSTAT
(Ekterly—Kalvista Pharmaceuticals)
Drug class: Ekterly is a plasma kallikrein inhibitor.
Indication: Ekterly is indicated for the treatment of acute attacks of hereditary angioedema in adult and pediatric patients aged 12 years and older.
Recommended dosage and administration: The recommended dosage is one dose of 600 mg (2 tablets) taken orally at the earliest recognition of an hereditary angioedema attack. A second dose of 600 mg may be taken 3 hours after the first dose if response is inadequate, or if symptoms worsen or recur. The maximum recommended dosage is 1,200 mg in any 24-hour period.
Common adverse effects: The most common adverse reaction is headache.
Warnings and precautions: Avoid use with strong CYP3A3 inhibitors. Patients taking moderate CYP3A4 inhibitors should take one 300-mg dose. A second dose of 300 mg may be taken at least 3 hours after the first dose if response is inadequate, or if symptoms worsen or recur. Avoid use with moderate or strong CYP3A4 inducers. Avoid use of Ekterly in patients with severe hepatic impairment. Patients with moderate hepatic impairment should take one 300-mg dose. A second dose of 300 mg may be taken at least 3 hours after the first dose if response is inadequate, or if symptoms worsen or recur.
New indications
LENACAPAVIR
(Yeztugo—Gilead Sciences)
Drug class: Yeztugo is a HIV-1 capsid inhibitor.
Indication: Yeztugo is indicated for PrEP to reduce the risk of sexually acquired HIV-1 in adults and adolescents weighing at least 35 kg who are at risk for HIV-1 acquisition. Individuals must have a negative HIV-1 test prior to initiating Yeztugo.
Recommended dosage and administration: All individuals should be screened for HIV-1 infection prior to initiating, prior to each injection, and additionally as clinically appropriate. The dosing schedule is comprised of initiation dosing followed by once every 6-months continuation injection dosing. Tablets may be taken without regard to food. On day 1, the recommended dose is 927 mg by S.C. injection and 600 mg orally. On day 2, the recommended dose is 600 mg orally. Continuation dosing is 927 mg by S.C. injection every 6 months from the date of the last injection (+/- 2 weeks). If the scheduled injection is anticipated to be delayed by more than 2 weeks, Yeztugo tablets may be used on an interim basis, and for up to 6 months if needed, until injections resume. The dosing schedule for delayed injection is 300 mg orally once every 7 days. If more than 28 weeks have elapsed since the last injection and tablets have not been taken, restart initiation from day 1 if clinically appropriate. Dosage modifications are recommended when initiating strong or moderate CYP3A inducers.
Common adverse effects: The most common adverse reactions are injection site reactions, headache, and nausea.
Boxed warning: Individuals must be tested for HIV-1 infection prior to initiating Yeztugo and with each subsequent injection of Yeztugo, using a test approved or cleared by FDA for the diagnosis of acute or primary HIV-1 infection. Drug-resistant HIV-1 variants have been identified with use of Yeztugo by individuals with undiagnosed HIV-1 infection. Do not initiate Yeztugo unless negative infection status is confirmed. Individuals who acquire HIV-1 while receiving Yeztugo must transition to a complete HIV-1 treatment regimen.
Other warnings and precautions: There is a potential risk of developing resistance to lenacapavir if an individual acquires HIV-1 either before or when receiving Yeztugo or following discontinuation of Yeztugo. Residual concentrations of lenacapavir may remain in systemic circulation for up to 12 months or longer. Improper administration has been associated with serious injection site reactions. Consult the full prescribing information for important drug interactions with Yeztugo.
New dosage forms
LISDEXAMFETAMINE DIMESYLATE ORAL SOLUTION
(Arynta—Azurity)
Drug class: Arynta is a central nervous system stimulant.
Indication: Arynta is indicated for the treatment of ADHD in adults and pediatric patients 6 years and older and moderate to severe binge eating disorder in adults. Pediatric patients with ADHD younger than 6 years old experienced more long-term weight loss than patients 6 years and older. Arynta is not indicated for weight loss. Use of other sympathomimetic drugs for weight loss has been associated with serious cardiovascular adverse events. The safety and effectiveness of Arynta for the treatment of obesity have not been established.
Recommended dosage and administration: In patients with ADHD, the recommended initial dose is 30 mg every morning, titrated by 10 mg or 20 mg weekly up to a total recommended dose of 30 mg to 70 mg per day. In patients with binge eating disorder, the recommended initial dose is 30 mg every morning, titrated up by 20 mg weekly, up to a total recommended dose of 50 mg to 70 mg daily. Prior to treatment, assess for the presence of cardiac disease. In patients with severe renal impairment, the maximum dose is 50 mg/day. In patients with end stage renal disease, the maximum dose is 30 mg/day.
Common adverse effects: The most common adverse reactions are anorexia, decreased appetite, decreased weight, diarrhea, dizziness, dry mouth, irritability, insomnia, nausea, upper abdominal pain, vomiting, increased heart rate, constipation, feeling jittery, and anxiety.
Boxed warning: Arynta has a high potential for abuse and misuse, which can lead to the development of an SUD, including addiction. Misuse and abuse of central nervous system stimulants, including Arynta, can result in overdose and death. Before prescribing Arynta, assess each patient’s risk for abuse, misuse, and addiction. Educate patients and their families about these risks, proper storage of the drug, and proper disposal of any unused drug. Throughout treatment, reassess each patient’s risk and frequently monitor for signs and symptoms or abuse, misuse, and addiction.
Other warnings and precautions: Arynta is contraindicated in patients with a known hypersensitivity to amphetamine products or other ingredients in Arynta and in use with monoamine oxidiase inhibitors or within 14 days of the last monoamine oxidase inhibitor dose. Avoid use in patients with known structural cardiac abnormalities, cardiomyopathy, serious cardiac arrhythmia, coronary artery disease, or other serious cardiac disease. Monitor BP and pulse. Prior to initiating Arynta, screen patients for risk factors for developing a maniac episode. If new psychotic or manic symptoms occur, consider discontinuing Arynta. Closely monitor growth in pediatric patients. Pediatric patients who are not growing or gaining height or weight as expected may need to have their treatment interrupted. Careful observation for digital changes is necessary during Arynta treatment. Further clinical evaluation may be appropriate for patients who develop signs or symptoms of peripheral vasculopathy. There is an increased risk of serotonin syndrome when Arynta is coadministered with serotonergic agents but also during overdosage situations. If it occurs, discontinue Arynta and initiate supportive treatment. Before initiating Arynta, assess the family history and clinically evaluate patients for tics or Tourette’s syndrome. Regularly monitor patients for the emergence or worsening of tics or Tourette’s syndrome. Discontinue treatment if clinically appropriate. Agents that alter urinary pH can alter blood levels of amphetamine. Acidifying agents decrease amphetamine blood levels while alkalinizing agents increase amphetamine blood levels. Adjust Arynta dose accordingly. Arynta may cause fetal harm, and breastfeeding is not recommended during treatment. ■
FDA to issue new commissioner’s national priority vouchers to companies
On June 17, 2025, FDA announced its Commissioner’s National Priority Voucher program. Drug developers can use these vouchers to participate in an FDA program that shortens the review time of a final drug application to 1–2 months, instead of the typical time frame of 10–12 months.
This new process includes a team-based review rather than using the standard review system of a drug application being sent to numerous FDA offices. The clinical information will be reviewed by a multidisciplinary team of physicians and scientists who will pre-review the application and then convene for a 1-day meeting.
Vouchers will be available to companies that are aligned with the national health priorities of addressing a health crisis in the United States delivering more innovative cures for the American people, addressing unmet public health needs, and increasing domestic drug manufacturing as a national security issue. If a product for which the voucher is used meets the legal requirements for accelerated approval, an accelerated approval may also be granted by FDA. The program will also include enhanced communication with the drug developing company throughout the entire review process. ■