LOUISVILLE, Ky.--(BUSINESS WIRE) --USWM, LLC (US WorldMeds) today announced that the U.S. Food and Drug Administration (FDA) has approved Iwilfin ™ (eflornithine) 192 mg tablets, a groundbreaking oral maintenance therapy for high-risk neuroblastoma. Iwilfin is indicated to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.

According to the American Cancer Society, 700-800 cases of neuroblastoma are diagnosed in the U.S. each year, with 90% of diagnoses coming before age 5. Over 50% of these cases are classified as high-risk. High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission. Approximately half of children with high-risk neuroblastoma do not survive beyond five years from diagnosis. Although existing treatments are effective in helping patients achieve remission, patients lack options to sustain it. Avoiding relapse is crucial to improving survival rates.

“We are thrilled to announce the FDA approval of Iwilfin , which provides a new and much-needed treatment option for children with high-risk neuroblastoma,” said Breck Jones, Chief Executive Officer of US WorldMeds. “The goal for treating these young patients is to prevent relapse, and advancing therapeutic options is critical to this mission. Iwilfin offers new hope and improved outcomes for these vulnerable children.”

The approval of Iwilfin is based on the results of a multi-site, single-arm, externally controlled study of children with high-risk neuroblastoma who received Iwilfin as maintenance therapy following standard of care treatment, including immunotherapy. The study demonstrated that the addition of Iwilfin improved event-free survival (EFS) and overall survival (OS) in patients with high-risk neuroblastoma. At four years following immunotherapy, EFS in the Iwilfin -treated patient group was 84% compared to 73% of patients in the external control group, and 96% of patients treated with Iwilfin were alive compared to 84% of external control patients. This corresponded to a 52% reduction in the risk of relapse and a 68% reduction in the risk of death. Across additional analyses to confirm the results of the externally controlled study design, the relapse risk reduction ranged from 57% to 41% and death risk reduction ranged from 71% to 55%.

Iwilfin is taken orally, with or without food, twice daily for two years. Iwilfin is generally well-tolerated, with side effects typically manageable through dose modifications. The most common side effects are hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. Important Safety Information can be found below.

US WorldMeds partnered with the Beat Childhood Cancer Research Consortium at Penn State University, which conducted the preclinical and clinical research to help advance this vital therapy. The Consortium represents a group of over 50 hospitals that offer collaboration through a network of childhood cancer clinical trials.

“Our partnerships were instrumental in bringing Iwilfin through the FDA registration process,” said Kristen Gullo, Vice President of Development and Regulatory Affairs at US WorldMeds. “We are thankful for the dedication of our partners, specifically the Beat Childhood Cancer Research Consortium, who work tirelessly to improve treatment outcomes for pediatric cancer patients. This FDA approval represents a beacon of hope for the high-risk neuroblastoma community and a significant step forward in the fight against this devastating disease.”

“After diligent efforts for the past decade, I am both humbled and overjoyed to see this product that holds the potential to evolve the standard of care for high-risk neuroblastoma come to fruition,” said Giselle Saulnier Sholler, MD, Chair and Founder, Beat Childhood Cancer Research Consortium and Division Chief of Pediatric Hematology and Oncology at Penn State Health Children’s Hospital. “I extend my deepest gratitude to every individual and institution that contributed to this extraordinary endeavor. The unwavering commitment of the sites involved and the incredible resilience of the families who entrusted us with their children's well-being have been the driving forces behind this milestone. Their commitment has not only propelled us forward in our mission but has also paved the way for a brighter future for children facing the challenges of cancer.”

Iwilfin will be available to patients in the United States in the coming weeks. To learn more about Iwilfin , visit www.Iwilfin .com.

Important Safety Information for Iwilfin (eflornithine) tablets

Before you take Iwilfin , tell your healthcare provider about your medical conditions, including if you have hearing problems, if you are pregnant or may become pregnant, or if you are breastfeeding.

If you or your partner are able to become pregnant, use effective birth control during treatment with Iwilfin and for one week after the last dose. Tell your healthcare provider right away if you or your partner become pregnant during treatment. Do not breastfeed during treatment with Iwilfin and for one week after the last dose.

Iwilfin can cause low blood cell counts. Tell your healthcare provider right away if you develop symptoms of low blood cell counts, including fever, easy bruising or bleeding, blood in your urine or stool, feeling unusually tired or weak, shortness of breath, or chills or shivering.

Iwilfin can cause liver problems. Tell your healthcare provider right away if you develop symptoms of liver problems, including your skin or white of your eyes turning yellow; dark urine; light-colored stools; nausea or vomiting; easy bruising or bleeding; loss of appetite; or pain, aching, or tenderness on the right side of your abdomen.

Hearing loss is common during treatment with Iwilfin and can be serious. Some people have needed to use hearing aids. Tell your healthcare provider right away if you get ringing in your ears or any new or worsening hearing loss.

What are the possible side effects of Iwilfin ?

The most common side effects of Iwilfin include ear infection; diarrhea; cough; sinus infection; pneumonia; upper respiratory tract infection; pink eye; vomiting; stuffy, runny, itchy nose or sneezing; and fever; skin infection; and urinary tract infection.

These are not all the possible side effects of Iwilfin . Contact your healthcare provider for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

About US WorldMeds

US WorldMeds is a privately held specialty pharmaceutical company that develops, licenses, and markets unique healthcare products designed to improve the lives of patients with challenging conditions and unmet medical needs. More information on US WorldMeds can be found at www.usworldmeds.com.

Iwilfin is a trademark of USWM, LLC

US WORLDMEDS is a registered trademark of USWM, LLC

Source: US WorldMeds

Posted: December 2023