PDR Drug Update - June 2026

Helping Improve Survival in Pediatric Menkes Disease

Menkes disease is a rare X-linked recessive pediatric disorder caused by mutations in the ATP7A gene, which is responsible for copper transport throughout the body. Disruption of ATP7A function results in systemic copper deficiency and reduced activity of copper-dependent enzymes involved in neurologic development, connective tissue formation, and energy metabolism. The disorder typically presents during infancy and primarily affects male patients. Clinical manifestations commonly include hypotonia, seizures, deterioration of the nervous system, developmental delay, intellectual disability, failure to thrive, sagging facial features, and characteristic sparse, kinky, or depigmented hair. Connective tissue, skeletal, vascular, and gastrointestinal abnormalities may also occur. Diagnosis is based on clinical findings such as low serum copper and ceruloplasmin concentrations, as well as results from procedures such as skin biopsies, ultrasounds, urine tests, CT scans, and X-rays. Genetic testing may also be used to confirm the diagnosis when available.

Menkes disease is progressive, and neurologic injury begins early in infancy, which contributes to a significant mortality rate, especially in untreated patients. Management of Menkes disease is multidisciplinary. Care typically includes pain medications, seizure management, nutritional support for feeding difficulties, and respiratory care for infections or related complications. Physical and occupational therapy may also be incorporated as part of developmental support. Off-label copper salt injections have also been used for treatment, with some success.

Zycubo (copper histidinate) was approved by the FDA in January 2026 as the first FDA-approved therapy for pediatric patients with Menkes disease. Zycubo is administered as a subcutaneous copper replacement therapy and is intended to increase systemic copper levels and bypass impaired intestinal copper transport. Dosage varies based on age, with the recommended dosage being 1.45 mg administered subcutaneously twice daily for patients less than 1 year old, and once daily for patients 1 to 17 years old.

FDA approval was based on data from two open-label, single-arm clinical studies evaluating pediatric patients with Menkes disease treated for up to three years. Outcomes were compared with external untreated control groups. The studies included 66 treated patients and 17 untreated patients. Treatment initiated within four weeks of birth was associated with a 78% reduction in mortality compared with untreated controls. Later initiation was also associated with improved survival, although outcomes appeared to be better with earlier treatment. Some treated patients survived beyond six years of age, whereas survival beyond three years of age is uncommon in untreated patients with Menkes disease.

Patients receiving Zycubo should be monitored for copper accumulation during treatment. Common adverse reactions reported include pneumonia, infections, respiratory failure, seizures, hemorrhages, hypotension, vomiting, tachycardia, pyrexia, anemia, volume depletion, fractures, dyspnea, transaminases elevation, diarrhea, and local injection-site reactions.

Menkes disease remains a rare pediatric genetic disorder with limited treatment options, and early recognition and intervention remain important due to early-onset neurologic injury, even with the FDA approval of Zycubo. Stay informed about emergent drug information, including further Menkes disease-related approvals, 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

Baxfendy

The FDA has approved Baxfendy (baxdrostat) as a first-in-class aldosterone synthase inhibitor for the treatment of hypertension in combination with other antihypertensive medications, to lower blood pressure in adults who are not adequately controlled. Learn more about Baxfendy

Beqalzi

The FDA has approved Beqalzi (sonrotoclax) for the treatment of adult patients with relapsed or refractory mantle cell lymphoma, after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase inhibitor. Learn more about Beqalzi

Idvynso

The FDA has approved Idvynso (doravirine/islatravir) for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in those who are virologically suppressed on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine. Learn more about Idvynso

Immgolis

The FDA has approved Immgolis (golimumab-sldi), a biosimilar to Simponi (golimumab), for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) in combination with methotrexate, and adult patients with moderately to severely active ulcerative colitis. The FDA also approved Immgolis Intri (golimumab-sldi), a biosimilar to Simponi Aria (golimumab), for the treatment of adults with moderately to severely active RA in combination with methotrexate. Learn more about Immgolis

Langlara

The FDA has approved Langlara (insulin glargine-aldy) as a biosimilar to Lantus (insulin glargine), for the treatment of adults and pediatric patients with type 1 diabetes mellitus and adults with type 2 diabetes mellitus. Learn more about Langlara

Otarmeni

The FDA has approved Otarmeni (lunsotogene parvec-cwha) for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 decibel hearing level) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. Learn more about Otarmeni

Trimbow

The FDA has approved Trimbow (beclomethasone dipropionate/formoterol fumarate/glycopyrrolate) for the maintenance treatment of asthma in adults. Trimbow delivers three active ingredients in a single device. Learn more about Trimbow

Veppanu

The FDA has approved Veppanu (vepdegestrant) for the treatment of adults with estrogen receptor-positive/human epidermal growth factor receptor 2-negative, estrogen receptor 1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy. Learn more about Veppanu

FDA New Indications

Asceniv

The FDA has approved an expanded indication for Asceniv (immune globulin intravenous, human – slra 10% liquid), expanding the primary humoral immunodeficiency indication to pediatric patients aged 2 years and older. Previously, the indication for Asceniv was restricted to patients aged 12 years and older. Learn more about Asceniv

Auvelity

The FDA has approved Auvelity (dextromethorphan HBr and bupropion HCl) for the treatment of agitation associated with dementia due to Alzheimer’s disease. Learn more about Auvelity

Bizengri

The FDA has approved Bizengri (zenocutuzumab-zbco) for the treatment of adults with advanced, unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 gene fusion with disease progression on or after prior systemic therapy. Learn more about Bizengri

Breztri

The FDA has approved Breztri Aerosphere (budesonide/glycopyrrolate/formoterol fumarate) for the maintenance treatment of asthma in adult and pediatric patients aged 12 years and older. Learn more about Breztri

Caplyta

The FDA has approved Caplyta (lumateperone) for the prevention of relapse in schizophrenia based on long-term data evaluating the drug’s safety and efficacy. Learn more about Caplyta

Dupixent

The FDA has approved Dupixent (dupilumab) for the treatment of children aged two to 11 years with chronic spontaneous urticaria (CSU) who remain symptomatic despite histamine-1 antihistamine treatment. This expands the previous approval for Dupixent in adults and adolescents aged 12 years and older with CSU. Learn more about Dupixent

Enhertu

The FDA has approved Enhertu (fam-trastuzumab deruxtecan-nxki) for two new breast cancer indications in the neoadjuvant and adjuvant settings of patients with HER2-positive early breast cancer. In the neoadjuvant setting, Enhertu followed by a taxane, trastuzumab, and pertuzumab has been approved for the treatment of adult patients with HER2-positive stage 2 or stage 3 breast cancer. In the adjuvant setting, Enhertu has been approved for the treatment of adult patients with HER2-positive breast cancer who have residual invasive disease following neoadjuvant trastuzumab (with or without pertuzumab) and taxane-based treatment. Learn more about Enhertu

Fasenra

The FDA has approved Fasenra (benralizumab) for the treatment of adult and pediatric patients aged 12 years and older with hypereosinophilic syndrome without an identifiable non-hematologic secondary cause. Learn more about Fasenra

Jakafi XR

The FDA has approved Jakafi XR (ruxolitinib) extended-release tablets for the treatment of adults with intermediate- or high-risk myelofibrosis; adults with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea; as well as adults and children aged 12 years and older with steroid-refractory acute graft-versus-host disease (GVHD) or chronic GVHD after failure of one or two lines of systemic therapy. Learn more about Jakafi XR

Ocrevus

The FDA has approved Ocrevus (ocrelizumab) intravenous infusion for the treatment of relapsing-remitting multiple sclerosis in pediatric patients aged 10 years and older who weigh 55 pounds or more. Learn more about Ocrevus

Saphnelo

The FDA has approved Saphnelo (anifrolumab) for self-administration as a once-weekly autoinjector, the Saphnelo Pen, for the treatment of adult patients with systemic lupus erythematosus on top of standard therapy. Learn more about Saphnelo

Stelara

The FDA has approved Stelara (ustekinumab) for the treatment of patients two years and older with moderately to severely active Crohn’s disease. Learn more about Stelara

Tecentriq

The FDA has approved Tecentriq (atezolizumab) and Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs) as an adjuvant treatment for adult patients with muscle-invasive bladder cancer who have circulating tumor DNA molecular residual disease after cystectomy. Learn more about Tecentriq

Tzield

The FDA has approved an expanded indication for Tzield (teplizumab-mzwv), extending the age range from 8 years and older to as young as 1 year to delay the onset of stage 3 type 1 diabetes (T1D) in patients diagnosed with stage 2 T1D. Learn more about Tzield

Vyvgart

The FDA has approved a label expansion for Vyvgart (efgartigimod alfa-fcab) and Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of adult patients with generalized myasthenia gravis. The approval expands Vyvgart’s indication to include all serotypes of adult patients living with gMG – anti-AChR-Ab positive, anti-MuSK-Ab positive, anti-LRP4-Ab positive, and triple seronegative. Learn more about Vyvgart

FDA New Combinations

Inqovi Combination Regimen

The FDA has approved Inqovi (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia who are 75 years or older or who are ineligible for intensive induction chemotherapy. Inqovi in combination with venetoclax is the first and only all-oral combination treatment regimen approved for this patient population. Learn more about Inqovi

FDA Recalls

B. Braun Lactated Ringer’s Injection

B. Braun Medical Inc. is voluntarily recalling two lots of Lactated Ringer’s Injection, E7500, 1L, to the hospital/healthcare facility level. The product has been found to have particulate matter in solution. Learn more about the Lactated Ringer’s injection recall

Pharmacal Eczema Cream

Pharmacal is recalling one lot of MG217 Multi-symptom Treatment Cream & Skin Protectant Eczema Cream, 6oz tube, to the consumer level. The product has been found to be contaminated with Staphylococcus aureus. Learn more about the eczema cream recall

Sun Pharmaceutical Doxorubicin HCl Liposome Injection

Sun Pharma is voluntarily recalling within the US to the hospital/user level, one batch of DOXOrubicin Hydrochloride Liposome Injection 50mg/25mL, Lot # HAG2581B, Expiration 05/2027 (675 vials). The single batch of 675 vials is being recalled due to the detection of glass particles in some vials during production. Learn more about the doxorubicin HCl liposome injection recall

FDA Alerts

Amazon Basic Care Levonorgestrel

The FDA is notifying consumers not to use expired Amazon Basic Care Levonorgestrel 1.5mg. The FDA is aware that several lots of this product were delivered to purchasers past the labeled expiration date. The products were distributed between January 24, 2025 and April 29, 2026. Consumers should check the expiration date on the product label and appropriately dispose of it immediately if that date has passed. Consumers who used this expired product should also contact their healthcare professional to determine the best course of action. Learn more about Amazon Basic Care Levonorgestrel

June 2026