PDR Drug Update - July 2026

Breakthrough Treatment for Otoferlin-Related Hearing Loss

Genetic causes account for approximately half of all cases of hearing loss present at birth, making inherited deafness one of the most common congenital sensory disorders worldwide. Among the many genes associated with congenital hearing loss, variants in the OTOF gene, which encodes the protein otoferlin, present a distinct and clinically important cause of profound sensorineural hearing loss. Although OTOF-related hearing loss accounts for only an estimated 2% to 8% of cases of inherited non-syndromic hearing loss, it has become the focus of groundbreaking advances in gene therapy that may fundamentally alter the treatment landscape.

Congenital hearing loss affects approximately 1 to 2 per 1,000 newborns globally, and genetic factors contribute to up to 60% of these cases. OTOF-related hearing loss is an ultra-rare disorder caused by biallelic pathogenic variants in the OTOF gene, meaning affected individuals receive a disease-causing variant from each parent. The OTOF gene encodes otoferlin, a protein that plays a critical role in auditory neurotransmission. Otoferlin is expressed primarily in the inner hair cells of the cochlea, the spiral-shaped structure of the inner ear. The absence or dysfunction of otoferlin prevents effective signal transmission from inner hair cells to the auditory nerve. As a result, sound is converted into electrical signals but fails to reach the brain. This condition is often classified as a form of auditory neuropathy and is associated with severe-to-profound congenital hearing loss, typically exceeding 70 to 90 decibels. Delayed diagnosis and intervention are common, which can significantly affect speech acquisition, language development, academic achievement, cognitive development, and literacy.

Until recently, treatment options for OTOF-related hearing loss were limited to assistive technologies such as cochlear implants. By bypassing the dysfunctional synapse and directly stimulating the auditory nerve, cochlear implants can provide substantial hearing benefit; however, they cannot address the underlying genetic defect. Patients may continue to experience difficulty with speech recognition in noisy environments, music appreciation, sound localization, and subtle auditory discrimination. In addition, cochlear implantation requires dependence on implanted hardware and ongoing device management. These limitations have created a significant unmet need for therapies that restore the natural auditory pathway rather than simply bypassing it.

OTOF-related hearing loss is particularly well suited for gene therapy because it results from the dysfunction of a single gene while the underlying auditory structures remain intact. The recent approval of Otarmeni (lunsotogene parvec-cwha) marks a historic milestone as the first FDA-approved gene therapy for a genetic form of hearing loss. It is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency greater than 90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. Otarmeni is an adeno-associated virus (AAV)-based gene therapy designed to deliver a functional copy of the OTOF gene directly to cochlear inner hair cells. The treatment is administered via a single intracochlear infusion under general anesthesia using a surgical approach similar to cochlear implantation. Once delivered, the therapeutic gene enables inner hair cells to produce functional otoferlin, restoring synaptic transmission between the cochlear sensory cells and the auditory nerve. Clinical trials demonstrated encouraging results. Approximately 80% of participants receiving treatment experienced clinically meaningful hearing improvement within 24 weeks. After a longer period, approximately 42% of these participants achieved hearing levels within the normal range, including the ability to detect whispered speech. Improvements have been sustained for up to two years in ongoing follow-up studies.

One of the greatest technical challenges in developing this gene therapy was the unusually large size of the OTOF gene. Standard AAVs have a limited carrying capacity and cannot accommodate the entire OTOF coding sequence within a single vector. To overcome this obstacle, researchers developed an innovative dual-vector strategy. The OTOF gene was divided into two separate components: a 5′ segment and a 3′ segment. Each portion was packaged into its own AAV vector, creating a paired-delivery system capable of carrying the full genetic information. When a patient receives the therapy, both vector components are infused into the cochlea. There, the fragments recombine to generate a full-length OTOF DNA sequence. As related cellular function proceeds, restoration of otoferlin production re-establishes synaptic function and allows auditory signals to be transmitted normally from the cochlea to the brain. This split-gene, dual-AAV approach represents a significant innovation in gene therapy and provides a framework for treating other disorders caused by genes that exceed the carrying capacity of conventional viral vectors.

Although continued clinical follow-up will be necessary to confirm long-term durability and functional outcomes, Otarmeni's success represents a landmark achievement in genetic medicine. For patients with OTOF-related hearing loss, it offers the potential to restore natural auditory function. For the broader field of hearing research, it provides a foundation for future gene therapies targeting additional inherited forms of deafness. Stay informed about emergent drug information, including gene therapies, by exploring PDR by ConnectiveRx. You can also download the official PDR app, mobilePDR, available for free from your preferred app store.

FDA New Approvals

Ambelvist

The FDA has approved Ambelvist (gadoquatrane), a new, intravenous macrocyclic gadolinium-based contrast agent indicated for contrast-enhanced magnetic resonance imaging to detect and visualize lesions with abnormal vascularity in the central nervous system (CNS) and non-CNS body regions in adult and pediatric patients, including term neonates. Learn more about Ambelvist

Cavhanza

The FDA has approved Cavhanza (nilotinib) orally disintegrating tablets. The Cavhanza formulation is specifically designed to improve solubility and dissolution rate, enabling maintained bioavailability with concomitant use of acid-reducing agents without timing restrictions. Cavhanza is indicated for the treatment of: newly diagnosed adults with Ph+ CML in chronic phase; and adults with chronic and accelerated phase Ph+ CML with resistance or intolerance to prior therapy, including imatinib. Learn more about Cavhanza

Cypsedo

The FDA has approved Cypsedo (cipepofol) for induction of general anesthesia in adults undergoing surgery. Learn more about Cypsedo

Decnupaz

The FDA has approved Decnupaz (pivekimab sunirine-pvzy) for the treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm, an ultra-rare and aggressive hematologic malignancy with limited treatment options. Learn more about Decnupaz

Hepcludex

The FDA has approved Hepcludex (bulevirtide-gmod) 8.5mg for the treatment of adults living with chronic hepatitis delta virus infection. Learn more about Hepcludex

Ranluspec

The FDA has approved Ranluspec (ranibizumab-hkdz) as an interchangeable biosimilar referencing Lucentis. Ranluspec is the only interchangeable biosimilar ranibizumab approved in the US in both vials and pre-filled syringes. Learn more about Ranluspec

Utebzi

The FDA has approved Utebzi (tebipenem pivoxil) for the treatment of complicated urinary tract infections including pyelonephritis, caused by certain susceptible pathogens in adult patients who have limited or no alternative oral treatment options. Learn more about Utebzi

Xocova

The FDA has approved Xocova (ensitrelvir), an oral antiviral, for post-exposure prophylaxis of COVID‑19 in adults and adolescents 12 years of age and older following contact with an individual who has COVID-19. Learn more about Xocova

Zaynich

The FDA has approved Zaynich (cefepime/zidebactam), a novel intravenous antibiotic for the treatment of adults with complicated urinary tract infections, including pyelonephritis, caused by susceptible Gram-negative pathogens. Learn more about Zaynich

FDA New Indications

Afrezza

The FDA has approved Afrezza (insulin human) inhalation powder for use in children and adolescents aged 6 and older living with type 1 and type 2 diabetes. This approval expands Afrezza’s availability beyond adults, introducing a new mealtime insulin option for pediatric patients and caregivers. Learn more about Afrezza

Capvaxive

The FDA has approved an expanded indication for Capvaxive (pneumococcal 21-valent conjugate vaccine) to include children and adolescents aged 2 through 17 years who have completed a primary pediatric pneumococcal vaccination series and have one or more chronic medical conditions that put them at an increased risk for pneumococcal disease. Learn more about Capvaxive

Datroway

The FDA has approved Datroway (datopotamab deruxtecan) for the treatment of adult patients with unresectable or metastatic triple-negative breast cancer who are not candidates for PD-1/PD-L1 inhibitor therapy. Learn more about Datroway

Hympavzi

The FDA has approved an expanded indication for Hympavzi (marstacimab-hncq) to include the treatment of patients with hemophilia A or B 12 years and older with inhibitors and pediatric patients (ages 6 to 11 years) with or without inhibitors. Hympavzi is now indicated in the US for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 6 years of age and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) with or without factor IX inhibitors. Learn more about Hympavzi

Linzess

The FDA has approved the use of Linzess (linaclotide) in pediatric patients 2 years of age and older with functional constipation (FC). Linzess was previously approved for pediatric patients 6 years and older with FC. With this expanded indication, Linzess is now available for children ages 2-5 years with FC and remains the only FDA-approved prescription therapy for pediatric FC. Learn more about Linzess

FDA New Combinations

Imfinzi Immunotherapy Combination

The FDA has approved Imfinzi (durvalumab) in combination with Bacillus Calmette-Guérin (BCG) induction and maintenance therapy for the treatment of adult patients with BCG-naïve, high-risk non-muscle-invasive bladder cancer. Learn more about Imfinzi immunotherapy combination

Keytruda, Keytruda Qlex with Welireg

The FDA has approved Keytruda (pembrolizumab) and Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph), each in combination with Welireg (belzutifan), for the adjuvant treatment of adult patients with renal cell carcinoma with a clear cell component at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. Learn more about Keytruda, Keytruda Qlex with Welireg

Truqap Treatment Combination

The FDA has approved Truqap (capivasertib) in combination with abiraterone and prednisone as targeted treatment for adult patients with PTEN-deficient metastatic androgen pathway modulation-naïve or sensitive prostate cancer, previously referred to as metastatic hormone-sensitive prostate cancer, as detected by an authorized test. Learn more about Truqap treatment combination

FDA New Dosing

Ebglyss

The FDA has approved a regimen of one maintenance dose every eight weeks of a single injection (250mg/2mL) of Ebglyss (lebrikizumab-lbkz) for subcutaneous use in adults and children 12 years of age and older who weigh at least 88 pounds (40kg) with moderate-to-severe atopic dermatitis. Ebglyss is already approved for a once-monthly maintenance dose, with long-term data showing durable disease control. Learn more about Ebglyss

FDA Recalls

BD ChloraPrep

BD (Becton, Dickinson and Company) is voluntarily recalling lot 4032183 of ChloraPrep Clear 1mL single sterile and lot 4073005 of FREPP clear 1.5mL applicators with paper lidding, to the consumer level. These products are being recalled due to fungal contamination under certain environmental conditions allowing the growth of Aspergillus penicillioides. Learn more about the ChloraPrep recall

Beekeeper’s Naturals Saline Nasal Spray

Beekeeper’s Naturals is voluntarily recalling lot # 5950, Exp. Date 02/2028 of Beekeeper’s Naturals Saline Nasal Spray, sold only through Amazon, to the consumer level. This lot, produced at a third-party manufacturer, tested above our acceptable microbiological limits for yeast and may contain Aspergillus spp. Learn more about the saline nasal spray recall

Haleon Gas-X

Haleon is voluntarily recalling four lots of Gas-X Extra Strength Softgels 125mg, 120 ct. and 72 ct. distributed on or about April 13, 2026, to the consumer level. The lots are being recalled due to potential contamination with a diluted propylene glycol-based coolant from a machine leakage during the packaging process. Learn more about the Gas-X recall

July 2026