FDA Approves Nuzolvence (zoliflodacin) for the Treatment of Uncomplicated Urogenital Gonorrhea in Adults and Adolescents

Innoviva Specialty Therapeutics, a subsidiary of Innoviva, Inc. (NASDAQ: INVA),   announced   the U.S. Food and Drug Administration (FDA)  approval of Nuzolvence (zoliflodacin) for oral suspension, a first-in-class, single-dose oral medication for the treatment of uncomplicated urogenital gonorrhea in adults and pediatric patients 12 years and older weighing at least 35 kg. The development of Nuzolvence was part of a private, not-for-profit collaboration with The Global Antibiotic Research and Development Partnership (GARDP), which sponsored and led the Phase 3 clinical trial that supported FDA approval.

“The FDA’s approval of Nuzolvence marks a pivotal moment for patients and the broader healthcare community managing gonorrhea infections. For the first time in decades, both patients and their healthcare providers will have a single-dose, oral treatment option for uncomplicated urogenital gonorrhea,” said David Altarac, M.D., Chief Medical Officer, Innoviva Specialty Therapeutics. “This achievement underscores our commitment to advancing innovative therapies for infectious diseases and fighting antimicrobial resistance. It highlights the strength of our development capabilities, as well as our collaboration with GARDP and the global scientific community.”

Gonorrhea is the second most common sexually transmitted bacterial infection worldwide, with more than 82 million new cases each year. In the United States alone, the Centers for Disease Control and Prevention (CDC) estimates that over 543,000 cases are reported each year and over 1 million incident cases occur annually,3 underscoring the significant public health impact. The highest rates are seen among sexually active men ages 20-24, though anyone who has unprotected sex can be at risk.4 Without timely treatment, gonorrhea can lead to serious and potentially permanent health complications.

"The decades-long absence of new gonorrhea treatments, combined with rising global antibiotic resistance, has created significant challenges in managing this common but potentially serious sexually transmitted infection," said Edward W. Hook III, M.D., Professor Emeritus of Medicine, University of Alabama at Birmingham, and lead investigator of the Phase 3 Nuzolvence trial. “In the pivotal Phase 3 study, Nuzolvence demonstrated non-inferiority compared to the current standard injectable therapy, including in infections caused by drug-resistant strains, while offering the convenience of a single oral dose.”

“A new antibiotic that does not require injection and can be used for patients who are allergic to penicillin or related drugs meets two important unmet needs in the treatment of gonorrhea,” added Dr. Hook.

The emergence and spread of global drug-resistant infections have led the World Health Organization (WHO) to identify antimicrobial resistance as one of the 10 most critical global health threats.5 The bacterium Neisseria gonorrhoeae has developed resistance to most classes of antibiotics used to treat these infections, including cephalosporins such as ceftriaxone, an injectable treatment, which is the currently recommended first line therapy for uncomplicated urogenital gonorrhea.

The Company plans to commercialize Nuzolvence in the second half of 2026, either in collaboration with a commercialization partner or independently.

https://en.wikipedia.org/wiki/Zoliflodacin

FDA Approves Nuzolvence (zoliflodacin) for the Treatment of Uncomplicated Urogenital Gonorrhea in Adults and Adolescents

FDA Approves Aqvesme (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases,  announced the U.S. Food and Drug Administration (FDA) approval of  Aqvesme (mitapivat), an oral pyruvate kinase (PK) activator, for the treatment of anemia in adults with alpha- or beta-thalassemia. With this approved indication, Aqvesme becomes the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

“Thalassemia is a debilitating disease that demands lifelong management and vigilant monitoring for many life-threatening complications, such as blood clots, heart disease, and liver disease. Despite its severity, treatments have historically been limited, leaving some patients without any options,” said Hanny Al-Samkari, M.D., Peggy S. Blitz Endowed Chair in Hematology/Oncology at Mass General Brigham Cancer Institute, Associate Professor at Harvard Medical School, and an investigator for the mitapivat thalassemia Phase 3 clinical program. “The ENERGIZE and ENERGIZE-T Phase 3 trial results demonstrate that Aqvesme can help address anemia, fatigue, and the need for regular transfusions – key challenges of the disease. Today’s FDA approval represents an important step forward for individuals with thalassemia.”

The FDA approval of Aqvesme in thalassemia is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. A total of 452 patients who are representative of the real-world thalassemia population were enrolled in the trials. The ENERGIZE and ENERGIZE-T Phase 3 trials met all primary and key secondary efficacy endpoints, demonstrating that Aqvesme improves hemolytic anemia and a key quality-of-life measure compared to placebo, including significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue.

“Today is a landmark moment for the thalassemia community, bringing forward an innovative, disease-modifying oral medicine to address the urgent needs of people living with this devastating rare blood disorder,” said Brian Goff, Chief Executive Officer, Agios. “With this approval, Aqvesme becomes the only medicine indicated for the treatment of anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. We are deeply grateful to the patients, caregivers, healthcare providers, and advocacy partners who participated in and supported our clinical trials and helped ensure that our efforts reflected their priorities. Our focus now is on ensuring a successful launch and delivering meaningful impact for the community.”

“This year marks 100 years since thalassemia was first described in medical literature, and the progress we’ve made as a community is truly extraordinary,” said Ralph Colasanti, National President, Cooley’s Anemia Foundation. “Innovative medicines like Aqvesme help make this progress possible, offering new hope to patients like me by helping to address the complex needs and challenges of this disease. This approval provides an important new treatment option for the thousands of adults living with thalassemia in the U.S., including those with non-transfusion-dependent thalassemia who previously had no approved alternatives.”

In the ENERGIZE and ENERGIZE-T Phase 3 trials, five patients receiving Aqvesme experienced adverse reactions suggestive of hepatocellular injury (HCI), with two of these patients requiring hospitalization. These adverse reactions occurred within the first six months of exposure, and liver tests improved upon discontinuation of Aqvesme. To mitigate the risk of HCI, Aqvesme is available only through the Aqvesme REMS, a Risk Evaluation and Mitigation Strategy (REMS) program approved by the FDA. The Aqvesme REMS program requires liver tests prior to the first Aqvesme dose, every four weeks thereafter for 24 weeks, and then as clinically indicated. It also includes education and certification requirements for patients, prescribing physicians, and pharmacists, which are common components of REMS programs.

Due to the Aqvesme REMS program, mitapivat will be marketed under the brand name Aqvesme in the U.S. for the thalassemia indication. Mitapivat will continue to be marketed as Pyrukynd® (mitapivat) in the U.S. for the PK deficiency indication, which does not require a REMS program. Outside the U.S., mitapivat will continue to be marketed as Pyrukynd for its PK deficiency and thalassemia indications in regions where it is approved, and will retain this name upon approval in regions currently under regulatory review.

Agios expects Aqvesme to be available in the U.S. in late January 2026, following implementation of the Aqvesme REMS program. For more information, visit Aqvesme.com.

About Thalassemia

Thalassemia is a rare, inherited blood disease that affects the production of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. The disease is categorized into two main types: alpha-thalassemia and beta-thalassemia, depending on which globin chain of the hemoglobin is affected. By disrupting hemoglobin production, thalassemia reduces the number of circulating red blood cells and shortens their lifespan, which leads to anemia, fatigue, and serious complications.

Some individuals with thalassemia require regular transfusions (classified as transfusion-dependent thalassemia), while others only need them intermittently (classified as non-transfusion-dependent thalassemia). All patients with thalassemia experience a significant disease burden, including comorbidities, reduced quality of life, and shortened life expectancy.

In the U.S., approximately 6,000 adult patients are diagnosed with thalassemia.

About ENERGIZE and ENERGIZE-T

ENERGIZE (NCT04770753) and ENERGIZE-T (NCT04770779) are global, double-blind, placebo-controlled Phase 3 trials evaluating the efficacy and safety of mitapivat in adults with alpha- or beta-thalassemia.

The ENERGIZE trial randomized 194 non-transfusion-dependent alpha- or beta-thalassemia patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was hemoglobin response, defined as an increase of ≥1.0 g/dL in average hemoglobin concentration from Week 12 through Week 24 compared with baseline. Key secondary endpoints included changes from baseline in average fatigue scores and in average hemoglobin concentration from Week 12 to Week 24. The trial also assessed safety and tolerability.

The ENERGIZE-T trial randomized 258 transfusion-dependent alpha- or beta-thalassemia patients 2:1 to receive either mitapivat 100 mg twice daily or placebo. The primary endpoint was transfusion reduction response, defined as a ≥50% reduction in transfused red blood cell (RBC) units with a reduction of ≥2 units of RBCs transfused in any consecutive 12-week period through Week 48 compared with baseline. Several transfusion reduction measures were included as key secondary endpoints, and achievement of transfusion independence was a secondary endpoint. The trial also assessed safety and tolerability.

For each trial, patients who completed the double-blind phase had the option to transition into a corresponding open-label extension phase, during which all patients receive mitapivat.

https://en.wikipedia.org/wiki/Mitapivat

FDA Approves Aqvesme (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

SQ Innovation, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved its drug-device combination Lasix ONYU (furosemide injection) for the treatment of edema (due to fluid overload) in adult patients with chronic heart failure. Lasix ONYU was developed to enable subcutaneous infusion of furosemide outside the healthcare setting for selected patients, as prescribed by a clinician without the need for a healthcare professional to administer the drug.

About 6.7 million Americans suffer from heart failure, with the prevalence expected to rise to 8.7 million by 2030. Heart failure is a leading cause of hospitalizations for individuals aged 65 and older with approximately 1.2 million hospitalizations per year [1].

Lasix ONYU consists of a novel high-concentration formulation of furosemide combined with a state-of-the-art small Infusor for treatment at home. The innovative design includes a reusable unit that can be used for 48 treatments and a plastic sterile single-use unit that is discarded after treatment. The two-component design reduces manufacturing complexity and cost, allowing the product to be offered at a different, more favorable price point which is expected to reduce barriers to widespread adoption.

“Lasix ONYU has the potential to be transformative in the care of patients experiencing worsening heart failure due to fluid overload,” said Pieter Muntendam, MD, founder, President and CEO of SQ Innovation. “Treating selected patients at home offers important benefits to patients, health systems and payors. We look forward to launching Lasix ONYU with leading health systems in the 4th quarter of 2025.”

Bioavailability and diuretic response were determined in a clinical study in which Lasix ONYU demonstrated complete bioavailability (112%) resulting in similar diuresis (115%) and natriuresis (117%) when compared to the same dose given by IV bolus. The biphasic delivery of furosemide by the Infusor resulted in a tempered diuretic response while IV bolus administration led to a shorter period of more intense diuresis. The results of the study were published in a leading cardiovascular journal [2].

“Heart failure is the most common serious medical condition in the U.S. and affects about one in four Americans during their lifetime. The number of patients affected is expected to double over the next 20 years and we currently already often lack adequate resources to take care of the 6.7 million patients affected presently – there are not enough beds, clinicians and funds”, said Dr. Javed Butler, Professor of Medicine at University of Mississippi and President, Baylor Scott and White Research Institute. “The only two actionable solutions now are more widespread adoption of guideline directed medical therapy (GDMT) and treating more patients at home with products such as subcutaneous diuretics instead of hospitalization for intravenous diuretics.”

“Decongestion through use of IV diuretics has been the cornerstone of treatment for reducing edema and hypervolemia in heart failure patients for over five decades,” stated S. Craig Thomas, Immediate Past President of the American Association of Heart Failure Nurses (AAHFN), an organization dedicated to advancing nursing education, clinical practice, and research to improve outcomes for heart failure patients. “The availability of accessible, affordable, and novel options that do not require the presence of a healthcare professional allows for transformative new clinical care-delivery. This means patients who now would typically need to be hospitalized for several days of IV treatment can instead remain home, supported by periodic or remote monitoring. The significance of this shift away from inpatient care for patients, hospitals, and payers cannot be overstated.”

Starting this quarter, Lasix ONYU will be available from leading pharmaceutical distributors enabling timely availability at participating medical facilities and affiliated retail pharmacies.

SQ Innovation is hosting a Conference Call and Webcast on Thursday October 9, at 4:30pm ET to introduce the product and answer questions from the community. Participating in the conference call will be:

Pieter Muntendam, MD, President and CEO of SQ Innovation

Mustafa M. Ahmed, MD, Professor of Medicine, Section Chief, Heart Failure, University of Florida Health, Gainesville, FL

Craig Thomas, Nurse Practitioner, Advanced Heart Failure Center, University of Virginia Health System, Charlottesville, VA and Immediate Past President American Association of Heart Failure Nurses (AAHFN)

https://en.wikipedia.org/wiki/Furosemide

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

FDA Approves Onapgo (apomorphine hydrochloride) for the Treatment of Motor Fluctuations in Adults with Advanced Parkinson’s Disease

Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, announced the U.S. Food and Drug Administration (FDA) approval of  Onapgo (apomorphine hydrochloride) injection, formerly known as SPN-830, as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson’s disease (PD). Supernus will make Onapgo available in the second quarter of 2025 with a support team of experts, including a robust nurse education program, and access support at launch.

“Continuous subcutaneous apomorphine infusion already has a proven and established 30-year history in Europe, where it has helped deliver more consistent control of motor fluctuations for thousands of patients,” said Rajesh Pahwa, M.D., Laverne and Joyce Rider Professor of Neurology at the University of Kansas School of Medicine, Director of the Movement Disorder Program at The University of Kansas Health System, and a clinical trial investigator for Onapgo. “In a clinical trial in Europe, patients treated with Onapgo experienced a significant reduction in daily OFF time and a similar significant increase in GOOD ON time. Today’s approval of Onapgo means patients in the U.S. who are not responding well to their current treatment regimen, including levodopa, will now have the option of using a small and lightweight wearable device to deliver a continuous infusion without the need for an invasive surgical procedure.”

The approval is based on results from a Phase 3, 12-week, multicenter, parallel-group, double-blind, randomized, placebo-controlled study (N=107) evaluating the efficacy and safety of Onapgo. The primary efficacy endpoint was the mean change in total daily OFF time assessed from baseline to the end of the 12-week treatment period based on patient diaries. The key secondary endpoints were the mean change in daily GOOD ON time, which was defined as ON time without troublesome dyskinesia, and Patient Global Impression of Change (PGIC).1

“Onapgo represents a novel approach for adults with Parkinson’s disease who are experiencing motor fluctuations,” said Jack Khattar, President and CEO of Supernus Pharmaceuticals. “Supernus’ significant experience in CNS has fueled the success of more than eight widely recognized products in CNS and other therapeutic categories. The addition of Onapgo demonstrates our continued commitment to developing novel alternatives to manage Parkinson’s disease and other neurological conditions.”

"As Parkinson’s disease progresses, levodopa treatment often becomes less effective at delivering consistent motor control in part due to GI dysmotility, variable absorption of oral medication, and the resulting pulsatile stimulation of dopamine pathways in the brain," said Stuart Isaacson, M.D., Director of Parkinson’s Disease and Movement Disorders Center of Boca Raton, Florida, and a clinical trial investigator for Onapgo. "With Onapgo, the continuous infusion of apomorphine directly stimulates postsynaptic dopamine receptors with no metabolic conversion needed. In addition, the subcutaneous delivery of apomorphine bypasses the GI tract and enters the brain, which can allow for more predictable symptom improvement."

“As the motor symptoms of Parkinson’s disease worsen over time, patients report alternating states between ON when their medication is working, and OFF when it’s not working optimally,” said Andrea Merriam, CEO of the Parkinson & Movement Disorder Alliance. “These on-again, off-again changes are disruptive and can happen at any time, which is why consistent daily control of OFF time is key to improving how patients feel and move. For many, continuous treatment options like Onapgo can help to make days with Parkinson’s more predictable.”

About the Phase 3 Study

During the Phase 3 study, Onapgo significantly reduced the amount of daily OFF time at 12 weeks from baseline (p=0.0114), with Onapgo-treated patients (n=53) experiencing a 2.6-hour reduction compared to placebo (n=51) with 0.9 hours. The reduction in daily OFF time was accompanied by a similar significant increase in daily GOOD ON time (2.8 hours for Onapgo-treated patients compared to 1.1 hours for the placebo group; p=0.0188).1* In addition, numerically greater improvements in daily OFF time and daily GOOD ON time were seen as early as week 1 and were maintained throughout all measured timepoints. Additionally, Onapgo-treated patients more frequently reported improvement in their state of general health compared with placebo-treated patients (PGIC: 79% vs. 24%; p<0.0001). The most common adverse events (≥10% incidence) were infusion-site nodule, nausea, somnolence, infusion-site erythema, dyskinesia, headache, and insomnia.1

About Parkinson’s disease

Nearly one million people in the U.S. and more than 10 million people worldwide are living with Parkinson’s disease, a progressive and chronic neurodegenerative disorder that can cause tremors, muscle rigidity, and difficulty with movement and balance. Patients may also experience dyskinesia, involuntary movements that can significantly interfere with daily activities.2 The disease impacts the central nervous system (e.g., the brain and spinal cord) and the peripheral nervous system, the network of nerves that support the limbs and the organs of the body (e.g., GI system including digestion, respiration, heart function, and blood pressure).3 While there is no known cure for PD, there are treatments available to help reduce symptoms.4 Patients treated with mainstay regimens may experience periods of GOOD ON time when medication treatment is working well, or OFF time when oral levodopa no longer provides symptom benefit and motor symptoms return.5 PD is the second most common neurodegenerative disorder of aging and the most common movement disorder.6

USE

Onapgo is a prescription medicine used to treat motor fluctuations (OFF episodes) in adults with advanced Parkinson’s disease (PD). It is not known if Onapgo is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Do not take Onapgo if you are:

taking certain medicines to treat nausea (ondansetron, granisetron, dolasetron, palonosetron) and alosetron. People taking ondansetron with apomorphine had very low blood pressure and lost consciousness (blacked out).

allergic to apomorphine or to any ingredients in Onapgo including sulfite. Sulfites can cause severe, life-threatening allergic reactions, especially in people with asthma.

Call your healthcare provider or get emergency help right away if you have any of the following symptoms of severe life-threatening allergic reaction:

hives • itching • rash • swelling (eyes, tongue, lips, or mouth) • chest pain • throat tightness • trouble breathing or swallowing.

Before you start using Onapgo, tell your healthcare provider about all of your medical conditions, including:

difficulty staying awake during the daytime • dizziness, fainting spells, or low blood pressure • asthma

allergies to any medicines containing sulfites • heart problems • a history of stroke or other brain problems

kidney problems • liver problems • a mental problem called a major psychotic disorder • drinking alcohol • if you are pregnant or plan to become pregnant, or breastfeeding or plan to breastfeed. It is not known if Onapgo will harm your unborn baby or pass into your breast milk.

Tell your healthcare provider about all the medicines you take, including prescription and non-prescription (over-the-counter) medicines, vitamins, and herbal supplements. Onapgo and certain other medicines may affect each other and cause serious side effects.

If you take nitroglycerin under your tongue (sublingual) while using Onapgo, your blood pressure may decrease and cause dizziness. If possible, lie down before taking it and then try to continue lying down for at least 45 minutes after.

What should I avoid while using Onapgo?

https://en.wikipedia.org/wiki/Apomorphine

https://go.drugbank.com/drugs/DB00714

FDA Approves Onapgo (apomorphine hydrochloride) for the Treatment of Motor Fluctuations in Adults with Advanced Parkinson’s Disease