PDR Drug Update - December 2024

Transformative Treatment for Degenerative Disease

The progressive and devastating disease Duchenne muscular dystrophy (DMD) brings with it severe limitations for the quality of life of those with the condition. It currently has no cure, but advances in pharmaceutical treatments offer new ways to continue the fight. The recent approval of the first nonsteroidal treatment for patients with all genetic variants of DMD is one such example that has the potential to improve outcomes and reduce suffering.

DMD is known to affect approximately 1 out of every 6000 males born. Due to its cause being a mutation on the X-chromosome, very few females have DMD. The disease progression involves weakening muscles over time due to a lack of the protein dystrophin. With lower levels of dystrophin, muscle cells have less protection against damage that comes from normal movement. This leads to inflammation and muscle wasting, resulting in loss of ambulation during adolescence with the possibility of respiratory and cardiovascular failure at any point. Digestive issues are also common due to muscle weakness in the digestive tract. Many who have the disease do not survive past their late twenties.

Diagnosis of DMD generally occurs after symptoms are noticed, such as if a child begins missing developmental milestones related to walking and speaking. Some children may also experience impairment in cognition or have learning disabilities. Genetic testing is used to identify if a patient has mutations evident in the dystrophin gene. If necessary, further testing via biopsy can also be performed to check the level of dystrophin a person has; the protein is usually undetectable or present in exceptionally low levels in those with DMD. The biopsy will also show the extent of tissue damage, death, and abnormal growth.

Treatment for DMD involves both pharmaceutical and non-pharmaceutical interventions. Physical and speech therapy are often introduced to improve or retain movement and motor skills. External aids such as leg or ankle braces may be used. Surgery is sometimes helpful for releasing tightened muscles and scoliosis. As respiratory problems emerge, attachment to a ventilator or performing a tracheostomy may be needed. Different categories of pharmaceutical therapies are recommended and serve to relieve varying issues. The corticosteroids prednisone and deflazacort help reduce inflammation and improve strength and other functions, which aids in extending patient survival.

Disease-modifying therapies designed to help remedy or supplement the missing dystrophin through genetic modification techniques include eteplirsen, golodirsen, viltolarsen, casimersen, and delandistrogene moxeparvovec. The most recent addition to the list of available treatments for DMD is Duvyzat (givinostat). FDA approved earlier in 2024 as the first nonsteroidal drug for the treatment of patients with all genetic variants of DMD, the small molecule is supplied as an oral suspension that contains the histone deacetylase (HDAC) inhibitor givinostat hydrochloride monohydrate. Givinostat’s ability to block HDAC enzymes suppresses some of the muscle loss that occurs with DMD, slowing the progression of the disease. The development of landmark medications like givinostat is vital to help improve the standard of care for people living with DMD. Stay informed about treatments for DMD and other genetic diseases by exploring PDR by ConnectiveRx. You can also stay current by using the official PDR app, mobilePDR, which is available for free from your preferred app store.

FDA New Approvals

Attruby

The FDA has approved Attruby (acoramidis), an orally administered near-complete (≥90%) stabilizer of transthyretin for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization. Learn more about Attruby

Aucatzyl

The FDA has approved Aucatzyl (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Learn more about Aucatzyl

Bizengri

The FDA has approved Bizengri (zenocutuzumab-zbco) for adults with pancreatic adenocarcinoma or non–small cell lung cancer that are advanced unresectable or metastatic and harbor a neuregulin 1 gene fusion who have disease progression on or after prior systemic therapy. Learn more about Bizengri

Emrosi

The FDA has approved Emrosi (minocycline HCl), extended-release capsules, 40mg, for the treatment of inflammatory lesions of rosacea in adults. Learn more about Emrosi

Danziten

The FDA has approved Danziten (nilotinib) tablets for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase and adult patients with chronic phase and acute phase resistant or intolerant to prior therapy that included imatinib. Learn more about Danziten

Imkeldi

The FDA has approved Imkeldi (imatinib) oral solution, the first oral liquid form of imatinib to treat certain forms of leukemia and other cancers. Imkeldi can help slow or prevent the growth of specific cancers, including chronic myeloid leukemia and acute lymphoblastic leukemia, myelodysplastic syndrome /myeloproliferative disease, and gastrointestinal tumors. Learn more about Imkeldi

Kebilidi

The FDA has approved Kebilidi (eladocagene exuparvovec-tneq) for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. Learn more about Kebilidi

Rapiblyk

The FDA has approved Rapiblyk (landiolol) in the hospital critical care setting for the treatment of supraventricular tachycardia (atrial fibrillation and atrial flutter). Learn more about Rapiblyk

Revuforj

The FDA has approved Revuforj (revumenib) for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients aged one year and older. Learn more about Revuforj

Yesintek

The FDA has approved Yesintek (ustekinumab-kfce), a biosimilar to the reference product, Stelara (ustekinumab). Yesintek, a monoclonal antibody, is approved for the treatment of Crohn's disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis. Learn more about Yesintek

Ziihera

The FDA has approved Ziihera (zanidatamab-hrii) 50mg/mL for injection for intravenous use for the treatment of adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer, as detected by an FDA-approved test. Learn more about Ziihera

FDA Labeling Changes

Fludarabine Phosphate

The FDA has approved updated drug labeling for fludarabine phosphate under Project Renewal. Fludarabine phosphate injection is now approved for the following new and revised indications: as a component of a combination regimen for the treatment of adults with B-cell chronic lymphocytic leukemia (CLL); and for the treatment of adults with B-cell CLL who have not responded to or whose disease has progressed during treatment with at least one alkylating-agent containing regimen. Additional labeling revisions include: the dosage regimen was revised and updated to include the recommended dosage for use in combination with cyclophosphamide and rituximab; and the boxed warning was removed and this information was incorporated in the Warnings and Precautions section. Learn more about fludarabine phosphate

FDA New Indications

Bimzelx

The FDA has approved Bimzelx (bimekizumab-bkzx) for the treatment of adults with moderate-to-severe hidradenitis suppurativa. Bimzelx is the first and only approved medicine designed to selectively inhibit interleukin 17F in addition to interleukin 17A. Learn more about Bimzelx

Imfinzi

The FDA has approved Imfinzi (durvalumab) for the treatment of adult patients with limited-stage small-cell lung cancer whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy. Learn more about Imfinzi

Jylamvo

The FDA has approved Jylamvo (methotrexate) to include the treatment of pediatric patients with acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis. Learn more about Jylamvo

Scemblix

The FDA has approved Scemblix (asciminib) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase. Learn more about Scemblix

FDA Recalls

Boulla Capsules

Boulla LLC is voluntarily recalling lot YZM240406, Exp 04/05/27, of ZoomMax Capsules and ZapMax Capsules packaged in 10-count blisters in a cardboard carton to the consumer level. The products were found to be tainted with sildenafil. Learn more about the Boulla capsules recall

Endo Clonazepam

Endo is expanding its previously announced voluntary recall of clonazepam orally disintegrating tablets, USP (C-IV), due to potential product carton strength mislabeling. Learn more about the Endo Clonazepam recall

Mxbbb Umary

Mxbbb is voluntarily recalling 1 lot of Umary acid hyaluronic, 30 caplets 850mg to the consumer level. The products have been found to have omeprazole and diclofenac. Learn more about the Mxbbb Umary recall

VitalityVita Capsules

VitalityVita is voluntarily recalling lot 230811, Exp 8/11/25, of VitalityXtra capsules and PeakMax capsules packaged in 10-count blisters in a cardboard carton to the consumer level. VitalityXtra was found to be tainted with sildenafil, and PeakMax capsules were found to be tainted with sildenafil and diclofenac. Learn more about the VitalityVita Capsules recall

FDA Alerts

Fullerton Wellness Compounded Drugs

The FDA is warning patients and healthcare professionals not to use drugs compounded and distributed by Fullerton Wellness. The FDA has concerns about the sterility of drugs distributed by Fullerton Wellness. Learn more about the Fullerton Wellness Compounded Drugs alert

Skin-Cap Aerosol Spray Products

The FDA warns consumers not to purchase or use Skin-Cap aerosol spray products because they may contain undisclosed steroids. Learn more about the Skin-Cap Aerosol Spray Products alert

December 2024