New Drug Express: FDA October Drug Approvals

So far in 2024, the FDA has approved several significant new drugs, including Eli Lilly’s Kisunla for Alzheimer’s treatment, Madrigal Pharmaceuticals’ Rezdiffra for metabolic dysfunction-associated fatty liver disease, and BMS’s KarXT, its first new schizophrenia drug in 35 years.

Several new drugs were also approved in October, with one company receiving a Complete Response Letter (CRL), and approval expected soon. Below is a summary of the FDA approvals in October.

Scemblix

Indication: FDA approved Novartis’ Scemblix for the expanded indication for the treatment of adult Philadelphia chromosome-positive chronic myelogenous leukemia (CML).

Scemblix is an oral tyrosine kinase inhibitor that works by blocking the BCR-ABL1 fusion protein, a known marker of certain leukemias. The drug was first approved three years ago for leukemia, gaining two indications. The first was for the treatment of Philadelphia chromosome-positive chronic-phase leukemia, with accelerated approval. The second fully approved indication is for the same patient group, additionally including those with the T315I mutation.

The expanded indication is supported by phase III ASC4FIRST study data, which showed that 68% of Scemblix patients reached the clinical endpoint, compared to 49% of patients treated with standard tyrosine kinase inhibitors such as imatinib and nilotinib. Scemblix is expected to reach peak sales of over $3 billion.

Jylamvo

Indication: Pediatric acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis

Shorla Oncology received an expanded approval for Jylamvo, now indicated for treating pediatric patients with acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA). According to the US-Ireland based pharmaceutical company, Jylamvo is the only oral liquid methotrexate approved for both adult and pediatric indications.

Jylamvo was first approved in November 2022 for treating adult patients with acute lymphoblastic leukemia, mycosis fungoides, relapsed or refractory non-Hodgkin’s lymphoma, rheumatoid arthritis, and severe psoriasis.

Shorla CEO Sharon Cunningham stated, “This approval marks a significant step forward in addressing unmet pediatric care needs in oncology and autoimmune diseases, following successful use in adult patients.”

Roxybond

Developer: Protega Pharmaceuticals

Indication: Severe pain

Protega Pharmaceuticals announced that the FDA approved ROXYBOND? 10mg Tablet (oxycodone hydrochloride immediate-release 10mg tablets) for the treatment of severe pain requiring opioid analgesics where alternative treatments are inadequate.

The FDA has already approved 10 opioid products with abuse-deterrent properties, but all except Roxybond are extended-release/long-acting (ER/LA) formulations. Roxybond is the first immediate-release (IR) opioid drug approved by the FDA with abuse-deterrent properties. It uses the SentryBond? abuse-deterrent technology, a multi-layered tablet designed to prevent misuse using physical and chemical methods. Test data show that Roxybond resists cutting, crushing, grinding, or tool destruction much more effectively compared to other immediate-release opioid painkillers.

Additionally, under multiple conditions, Roxybond tablets resist home or experimental solvents, thus offering resistance to chemical extraction. The viscous material in Roxybond also makes it difficult to inject through a needle and complicates solution preparation, effectively preventing intravenous and intranasal (snorting) abuse. Besides the FDA-approved 10mg tablet, ROXYBOND has also been approved in 5mg, 15mg, and 30mg tablets. The addition of ROXYBOND 10mg enhances the flexibility and precision of opioid pain management for doctors and patients.

Orlynvah

Indication: Urinary tract infections

The FDA approved Orlynvah for the treatment of uncomplicated urinary tract infections (uUTIs) in adult women caused by specific microorganisms (Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis), who have few or no alternative oral antibacterial treatment options. According to the press release, Orlynvah is the first oral penem-class antibiotic approved by the FDA and the second uUTI treatment approved in the past 20 years.

The approval of Orlynvah is supported by two phase III trials. In the pivotal late-stage REASSURE study, Iterum's drug demonstrated statistical non-inferiority in overall response compared to Augmentin (amoxicillin/clavulanate), and in the pivotal phase III SURE1 study, Orlynvah outperformed ciprofloxacin in patients with quinolone-resistant infections. The safety profile was similar between the treatment groups in both studies.

Abrysvo

Indication: Respiratory syncytial virus (RSV)

After the FDA expanded the label for Pfizer’s Abrysvo, young individuals at high risk of respiratory syncytial virus (RSV)-related lower respiratory tract disease now have a preventive option. According to Pfizer, Abrysvo is the first RSV vaccine approved for adults under 50 years of age.

The expanded label was supported by phase III MONeT trial results, which showed non-inferior immune responses in young individuals aged 60 and older, as disclosed by Pfizer in April. Abrysvo is also the only vaccine option for pregnant women between 32 and 36 weeks of gestation, aiming to protect infants from birth to 6 months.

Vyloy

Indication: Gastric or gastroesophageal junction cancer

After the FDA approved Astellas’ Vyloy, certain gastric cancer patients now have a new treatment option. According to Astellas, Vyloy became the first claudin18.2-targeted therapy approved in the U.S. for gastric or gastroesophageal junction cancer.

Vyloy was specifically approved as a first-line treatment for claudin18.2-positive HER2-negative tumors, in combination with fluoropyrimidine- and platinum-based chemotherapy.

The approval is supported by data from phase III SPOTLIGHT and GLOW studies, both of which met the primary endpoints for progression-free survival and key secondary endpoints for overall survival. In both studies, Vyloy demonstrated good tolerability, with the most common side effects being nausea, vomiting, and decreased appetite.

Vyalev

Indication: Parkinson’s disease

With the FDA approval of AbbVie’s Vyalev as the “first and only subcutaneous infusion therapy based on levodopa for motor fluctuations,” this new non-surgical option provides continuous delivery of levodopa for early, middle, and late-stage Parkinson’s disease patients.

The approval was based on a pivotal 12-week phase III trial assessing Vyalev’s efficacy in adult patients with advanced Parkinson’s disease, compared to oral immediate-release carbidopa/levodopa. In addition, a 52-week open-label study evaluated Vyalev’s long-term safety and efficacy.

The pivotal trial results showed that Vyalev-treated patients exhibited better improvement in motor fluctuations, with longer periods of symptom control and shorter periods of motor off time compared to oral medication. By week 12, the Vyalev group showed 2.72 more hours of motor symptom control, compared to 0.97 hours in the active control group.

Lumryz

Indication: Narcolepsy-related cataplexy or excessive daytime sleepiness in children

Avadel Pharmaceuticals’ Lumryz has now been approved for the treatment of cataplexy or excessive daytime sleepiness (EDS) in pediatric patients aged 7 and older with narcolepsy.

Lumryz is a bedtime extended-release sodium oxybate formulation that was initially approved by the FDA on May 1, 2023, for the treatment of cataplexy or EDS in adult narcolepsy patients. Its approval in adults was based on positive results from the phase III REST-ON trial, which showed highly statistically significant and clinically meaningful results compared to placebo on three primary endpoints (maintenance of wakefulness testing, clinical global impression-improvement, and weekly cataplexy attack rate). With pediatric approval, Lumryz also gained orphan drug exclusivity, lasting until October 16, 2031.

Hympavzi

Indication: Hemophilia A and B

The FDA approved Pfizer’s Hympavzi as the first once-weekly subcutaneous preventive injection for Hemophilia B in the U.S. Hympavzi is also the first treatment method authorized for both Hemophilia A and B using pre-filled pens or syringes.

The approval is based on data from the phase III BASIS trial, which involved nearly 190 patients with severe Hemophilia A or moderate-to-severe Hemophilia B. In this trial, Pfizer’s drug reduced bleeding by 92% in patients without inhibitors to factor VIII or IX, with the annual bleeding rate reduced by 35%.

Dasiglucagon

Indication: Congenital hyperinsulinism

Zealand Pharma received a Complete Response Letter (CRL) for the first part of its new drug application (NDA) for dasiglucagon, which is intended to prevent and treat hypoglycemia in pediatric patients with congenital hyperinsulinism (CHI) aged 7 days and older. The CRL raised no concerns regarding the clinical data or safety of dasiglucagon but requested additional analysis of existing CGM data to support its use after 3 weeks.

Zealand Pharma expects to submit the additional data by the end of 2024.