Monthly Archives: June 2023

FDA Approves Roctavian (valoctocogene roxaparvovec-rvox) Gene Therapy for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc., announced that the United States Food and Drug Administration (FDA) approved Roctavian™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. Roctavian was first approved by the European Medicines Agency in August 2022. Safety results for 134 patients have been reported over three years, demonstrating that ROCTAVIAN was well-tolerated.

Hemophilia A is a lifelong, genetic condition caused by a mutation in the gene responsible for producing a protein called FVIII, which is necessary for blood clotting. When severely deficient in amount, the condition puts people with hemophilia A at risk for painful and potentially life-threatening bleeds, which can occur spontaneously. With the current standard of care, individuals undergo lifelong preventative therapy, receiving infusions or injections at burdensome routine intervals to maintain enough clotting factor in the bloodstream to prevent bleeds. Roctavian is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own FVIII and thereby limit bleeding episodes.

As part of the development of Roctavian, BioMarin has worked with private and public payers in the U.S. in parallel to enable access, with the goal of ensuring that every eligible adult interested in Roctavian is able to receive treatment.

It is estimated that there are approximately 6,500 adults living with severe hemophilia A in the U.S. BioMarin expects approximately 2,500 of those adults to be eligible to receive Roctavian with this initial approval.

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The U.S. Food and Drug Administration (FDA) approved BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy for the treatment of adults with severe hemophilia A (congenital Factor VIII [FVIII] deficiency with FVIII activity.

Also on June 29, 2023, the FDA approved ARUP Laboratories’ AAV5 DetectCDx as a companion diagnostic to aid in the selection of adult patients eligible for treatment with Roctavian. Based on the label, BioMarin estimates that about 2500 patients may be eligible to receive Roctavian.

Roctavian consists of a viral vector carrying the F8 gene that encodes FVIII and is administered intravenously (IV) as a one-time dose. Roctavian is not intended for administration in women.

Due to its high cost, it has been recommended that payers implement prior authorization to reserve Roctavian’s use for patient populations most likely to benefit from this treatment.

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FDA Approves Ngenla (somatrogon-ghla) Long-Acting Once-Weekly Treatment for Pediatric Growth Hormone Deficiency

Pfizer Inc. and OPKO Health Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Ngenla (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. Ngenla is expected to become available for U.S. prescribing in August 2023.

Ngenla (somatrogon-ghla) is a human growth hormone that works by replacing the lack of growth hormone in the body. Ngenla is taken by injection just below the skin, administered via a device that allows for titration based on patient need. Compared to the growth hormone, GENOTROPIN (somatropin), its action in the body lasts longer, enabling weekly injections instead of daily.

Growth hormone deficiency (GHD) is a rare disease characterized by the inadequate secretion of the growth hormone somatropin from the pituitary gland, affecting one in approximately 4,000 to 10,000 children. Without treatment, children will have persistent growth attenuation, a short height in adulthood, and puberty may be delayed. Children living with GHD may also experience challenges in relation to their physical health and mental well-being.

The most common side effects of any prescription drug are gastrointestinal issues, including nausea, constipation, and diarrhea, because most drugs go through the digestive system to be absorbed. Other common effects include drowsiness, pain, and skin reactions. Serious side effects associated with Ngenla may include:

• Fluid retention.
• Glucose intolerance and diabetes mellitus.
• Hypoadrenalism.
• Hypothyroidism.
• Increased mortality in patients with acute critical illness.
• Increased risk of neoplasm.
• Intracranial hypertension.
• Progression of preexisting scoliosis.

Ngenla is approved for the treatment of pediatric GHD in more than 40 markets including Canada, Australia, Japan, and EU Member States.

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FDA Approves Lantidra (donislecel-jujn) Cellular Therapy to Treat Patients with Type 1 Diabetes

The U.S. Food and Drug Administration approved Lantidra, the first allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes. Lantidra is approved for the treatment of adults with type 1 diabetes who are unable to approach target glycated hemoglobin (average blood glucose levels) because of current repeated episodes of severe hypoglycemia (low blood sugar) despite intensive diabetes management and education.

Type 1 diabetes is a chronic autoimmune disease that requires lifelong care including requiring insulin, either through multiple daily injections or continuous infusion using a pump, every day to live. People with type 1 diabetes also perform blood glucose checks several times a day to guide the management of their diabetes.

Some people with type 1 diabetes have trouble managing the amount of insulin needed every day to prevent hyperglycemia (high blood sugar) without causing hypoglycemia. They may also develop hypoglycemia unawareness, where they are unable to detect their blood glucose is dropping and may not have a chance to treat themselves to prevent their blood glucose from further dropping. This makes it difficult to dose insulin. Lantidra provides a potential treatment option for these patients.

When Lantidra is infused into the body, specifically into the hepatic portal vein, it allows the islet beta cells to enter the bloodstream and reach the liver. Additionally, immunosuppressive medications are needed to maintain the transplanted islet cells’ viability.

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FDA Approves Rystiggo (rozanolixizumab-noli) for the Treatment of Adults with Generalized Myasthenia Gravis

UCB (Euronext Brussels: UCB), a global biopharmaceutical company, has announced Rystiggo (rozanolixizumab-noli) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

gMG is a rare, chronic, heterogeneous (phenotypic and pathogenic), unpredictable autoimmune disease characterized by dysfunction and damage at the neuromuscular junction (NMJ). Several factors are understood to be drivers of gMG disease pathology, including complement-cascade, immune cells, and pathogenic IgG autoantibodies. Pathogenic IgG autoantibodies can impair synaptic transmission at the NMJ by targeting specific proteins on the post-synaptic membrane. This disrupts the ability of the nerves to stimulate the muscle and results in a weaker contraction. gMG has a global prevalence of 100–350 cases per every 1 million people.

Rozanolixizumab-noli injection for subcutaneous infusion is a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor (FcRN), resulting in the reduction of circulating IgG. It is the only FDAapproved treatment in adults for both anti-AChR and anti-MuSK antibody-positive gMG, the two most common subtypes of gMG.

The most common side effects of RYSTIGGO include:
• headache
• infections
• diarrhea
• fever
• hypersensitivity reactions
• nausea

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FDA Approves Litfulo (ritlecitinib) for Adults and Adolescents With Severe Alopecia Areata

Pfizer Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved Litfulo (ritlecitinib), a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata. The approved recommended dose for Litfulo is 50 mg. It is the first and only treatment approved by the FDA for adolescents (12+) with severe alopecia areata.

Alopecia areata is an autoimmune disease characterized by patchy or complete hair loss on the scalp, face, or body. It has an underlying immuno-inflammatory pathogenesis and develops when the immune system attacks the body’s hair follicles, causing hair to fall out. This hair loss often occurs on the scalp, but it can also affect eyebrows, eyelashes, facial hair, and other areas of the body. Alopecia totalis (total scalp hair loss) and alopecia universalis (total body hair loss) are types of alopecia areata.

Impacting nearly 7 million people in the U.S. and approximately 147 million people globally, alopecia areata can affect people of any age, gender, race, or ethnicity and can cause considerable burden beyond hair loss. Nearly 20% of people with alopecia areata are diagnosed before the age of 18.

Litfulo is a kinase inhibitor which inhibits Janus kinase 3 (JAK3) and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases, and it will be available in the coming weeks. The most common adverse events reported in at least 4% of patients include headache, diarrhea, acne, rash, and urticaria.

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FDA Approves Suflave (polyethylene glycol 3350, sodium sulfate, potassium chloride, magnesium sulfate, and sodium chloride) Colonoscopy Preparation that Tastes Similar to a Sports Drink

Sebela Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted approval of Suflave™ (polyethylene glycol, sodium sulfate, potassium chloride, magnesium sulfate, and sodium chloride for oral solution), for colonoscopy preparation in adults. Suflave, a lowvolume, safe and effective colonoscopy preparation – with a taste similar to a lemon-lime sports drink – was developed and will be marketed by Braintree Laboratories, part of Sebela Pharmaceuticals.

Additionally, colonoscopy preparation products and processes are commonly cited as a significant patient barrier to colon cancer screening, reinforcing an unmet need among patients for better bowel preparation options. Tolerance of bowel preparation remains the largest deterrent for patients considering screening colonoscopy with 71% of patients saying it is the worst part of colonoscopy and 55% indicating it was the greatest deterrent to a future colonoscopy. Prep volume, palatability and patient education are three key factors in reducing prep hesitancy.

Suflave will be available by prescription to patients in the U.S. in early August.

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FDA Approves Elevidys (delandistrogene moxeparvovec-rokl) Gene Therapy to Treat Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc., announced U.S. Food and Drug Administration (FDA) accelerated approval of Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval based on expression of Elevidys micro-dystrophin observed in patients treated with Elevidys. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. The disease occurs due to a defective gene that results in absence of dystrophin, a protein that helps keep the body’s muscle cells intact. As a result of this genetic defect, individuals with DMD may have symptoms such as trouble walking and running, falling frequently, fatigue, learning disabilities/difficulties, heart issues as a result of impact on heart muscle functioning, and breathing problems due to weakening of respiratory muscles involved in lung function.

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin. This accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle.

The most common adverse reactions in clinical studies were vomiting, nausea, liver function test increased, pyrexia and thrombocytopenia.

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FDA Grants Full Approval for Blincyto (blinatumomab) to Treat Minimal Residual Disease-Positive B-Cell Precursor Acute Lymphoblastic Leukemia

• Conversion From Accelerated to Full Approval
• Reinforces Blincyto as Standard of Care for Patients With Minimal Residual Disease at Baseline After Remission

Amgen announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Blincyto (blinatumomab) for the treatment of adults and pediatric patients with CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%, based on additional data from two Phase 3 studies that were submitted. The approval converts Blincyto’s accelerated approval to a full approval.

Amgen continues to advance a robust development program for Blincyto, including studies aimed at treating patients with MRD-negative B-ALL, trials designed to minimize chemotherapy, and the clinical investigation of a subcutaneous formulation, all intended to help address remaining unmet needs for patients.

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FDA Approves Lodoco (colchicine) as the First Anti-Inflammatory Drug for Cardiovascular Disease

AGEPHA Pharma USA, LLC, has announced that, following a Priority Review, the U.S. Food and Drug Administration (FDA) has approved Lodoco® as the first anti-inflammatory atheroprotective cardiovascular treatment demonstrated to reduce the risk of myocardial infarction (MI), stroke, coronary revascularization, and cardiovascular death in adult patients with established atherosclerotic disease or with multiple risk factors for cardiovascular disease.

AGEPHA Pharma anticipates that Lodoco, which can reduce the risk of cardiac events in patients with established cardiovascular diseases by 31% on top of standard of care, will be available for prescription in the second half of 2023. The company has not determined an exact price but is planning to offer a patient assistance program to ensure that patients who otherwise could not afford the Lodoco will be able to have access to the drug.

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