VAFSEO®

by NewsUncategorized

FDA Approves Vafseo for the Treatment of Anemia due to Chronic Kidney Disease
in Adult Patients on Dialysis

AkebiaTherapeutics, Inc., announced that the U.S. Food and Drug Administration (FDA) has approved Vafseo® (vadadustat) Tablets for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Vafseo is a once-daily oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin to manage anemia. Vafseo is now approved in 37 countries.

Approximately 500,000 adult patients in the U.S. on dialysis suffer from anemia due to CKD, which may be associated with many adverse clinical outcomes. The burden of managing uncontrolled anemia in CKD patients can be substantial, both in terms of healthcare costs and the impact on patients, healthcare providers and caregivers.


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WINREVAIR™

by NewsUncategorized

FDA Approves Winrevair a First-in-Class Treatment for Adults with Pulmonary Arterial Hypertension

Merck announced that the U.S. Food and Drug Administration (FDA) has approved sotatercept-csrk (U.S. Brand Name: Winrevair™, for injection, 45mg, 60mg) for the treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group 1) to increase exercise capacity, improve WHO functional class (FC), and reduce the risk of clinical worsening events. Winrevair was previously granted Breakthrough Therapy Designation by the FDA. Winrevair is the first FDA-approved activin signaling inhibitor therapy for PAH, representing a new class of therapy that works by improving the balance between pro- and anti-proliferative signaling to regulate vascular cell proliferation underlying PAH.

The approval is based on the Phase 3 STELLAR trial, which compared Winrevair (n=163) to placebo (n=160), both in combination with background standard of care therapies in adult patients with PAH (WHO Group 1 FC II or III).

Healthcare providers should monitor hemoglobin and platelets before each dose of Winrevair for the first five doses, or longer if values are unstable, and periodically thereafter to determine if dose adjustments are required. Winrevair may increase hemoglobin and may lead to erythrocytosis, which if severe may increase the risk of thromboembolic events or hyperviscosity syndrome. Winrevair also may decrease platelet count and lead to severe thrombocytopenia, which may increase the risk of bleeding; thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Treatment should not be initiated if platelet count is <50,000/mm.

Winrevair is given once every three weeks by subcutaneous injection and may be administered by appropriate patients or caregivers with guidance, training, and follow-up from a healthcare provider. Healthcare providers and patients/caregivers should refer to the Instructions for Use for information on the proper preparation and administration of Winrevair. Merck estimates that Winrevair will be available for dispensing by select specialty pharmacies in the U.S. by the end of April 2024.

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OPSYNVI®

by NewsUncategorized

FDA Approves Opsynvi (macitentan and tadalafil) for Adults with Pulmonary Arterial Hypertension

Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has approved Opsynvi® – a single-tablet combination of macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) inhibitor – for the chronic treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group I) and WHO functional class (FC) IIIII. Opsynvi® may be used in patients with PAH who are treatment-naïve or who are already on an ERA, PDE5 inhibitor or both. Opsynvi® may be used in patients who are currently treated concomitantly with stable doses of macitentan 10mg and tadalafil 40mg (20mg x 2) as separate tablets.

With the approval, Johnson & Johnson now offers a PAH portfolio addressing all three foundational and guideline-recommended pathways – nitric oxide, endothelin, and prostacyclin. PAH is a rare, progressive, and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation that eventually leads to right heart failure. An estimated 500 to 1,000 new cases of PAH are diagnosed each year in the U.S., classifying the disease as a rare condition.

The 2022 European Society of Cardiology (ESC)/European Respiratory Society (ERS) clinical guidelines recommend initial combination therapy of an ERA and a PDE5 inhibitor for patients with idiopathic PAH, heritable drug-associated PAH, or PAH-associated with connective tissue disease without cardiopulmonary comorbidities at low or intermediate risk.

Individually, macitentan reduces the risk of clinical worsening events and hospitalization, and tadalafil improves exercise ability.

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DUVYZAT™

by NewsUncategorized

DA Approves Duvyzat for Duchenne Muscular Dystrophy

Italfarmaco S.p.A. announced that the U.S. Food and Drug Administration (FDA) has approved Duvyzat™ (givinostat), a novel histone deacetylase (HDAC) inhibitor, for the treatment of patients 6 years or older with Duchenne muscular dystrophy (DMD), a rare X-linked progressive and life-limiting neuromuscular condition with symptoms from early childhood. DMD is a severe neuromuscular genetic disease characterized by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. DMD is caused by mutations in the dystrophin gene that result in the absence of a functional dystrophin protein. Duvyzat, an oral suspension, received priority review, orphan drug, and rare pediatric disease designations from the FDA. A Marketing Authorization Application (MAA) for givinostat as a potential treatment for DMD has been submitted to the European Medicine Agency (EMA) and is currently under review.

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TRYVIO™

by NewsUncategorized

FDA Approves Tryvio for the Combination Treatment of Resistant Hypertension

Idorsia Pharmaceuticals U.S. Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Tryvio™ (aprocitentan) for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs. Lowering blood pressure reduces the risk of fatal and non-fatal cardiovascular events, primarily strokes and myocardial infarctions. The recommended dosage of Tryvio is 12.5mg orally once daily, with or without food.

Tryvio (aprocitentan) is an endothelin receptor antagonist that inhibits the binding of endothelin (ET)-1 to ETA and ETB receptors. The effects of ET-1 bear many similarities with the pathophysiology of hypertension, and ET-1 is a major driver of aldosterone production. Until the approval of Tryvio, no systemic antihypertensive medications targeted the ET pathway, as approved antihypertensive therapies focus on the regulation of salt and water (diuretics), antagonism of the renin–angiotensin–aldosterone (RAAS) system, reduction of influx of extracellular calcium into the cell (calcium channel blockers), sympatholytic activity (beta blockers, central alpha-agonist agents), or non-selective vasodilatory effects.

Tryvio is available only through a restricted program under a REMS called the Tryvio REMS because of the risk of embryo-fetal toxicity. Prescribers must be certified with the Tryvio REMS by enrolling and completing training. Pharmacies that dispense Tryvio must be certified with the Tryvio REMS.


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REZDIFFRA

by NewsUncategorized

FDA Grants Accelerated Approval for Rezdiffra for the Treatment of Patients with Noncirrhotic Nonalcoholic Steatohepatitis (NASH)

Madrigal Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Rezdiffra (resmetirom) in conjunction with diet and exercise for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH), with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). Continued approval for this indication may be contingent upon verification and description of clinical benefits in ongoing confirmatory trials.

Rezdiffra is a once-daily, oral THR-beta agonist designed to target key underlying causes of NASH. The accelerated approval of Rezdiffra was based on results from the Phase 3 MAESTRO-NASH trial, which was recently published in the New England Journal of Medicine.

The Rezdiffra prescribing information does not include a liver biopsy requirement for diagnosis. Rezdiffra should not be used in patients with decompensated cirrhosis.


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LENMELDY™

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FDA Approves Lenmeldy for Children with Metachromatic Leukodystrophy

Orchard Therapeutics/Kyowa Kirin has announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy™, formerly known as OTL-200, for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

Metachromatic leukodystrophy is a rare and severe genetic condition that affects the brain and nervous system. Metachromatic leukodystrophy is caused by a faulty gene, which means that the body does not make enough of an enzyme called arylsulfatase A (ARSA). That lack of ARSA causes fatty substances called sulfatides to build up in the brain and nerves, leading to problems with movement and thinking abilities, severe spasticity, seizures, and patients gradually losing the ability to move, talk, swallow, eat, and see.

Lenmeldy is a gene therapy used to treat of types of metachromatic leukodystrophy in children. Lenmeldy is made using the patient’s own stem cells that have been modified so that the body can make the ARSA enzyme to help stop the progression of MLD. Lenmeldy is given as a one-time infusion.


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TEVIMBRA

by NewsUncategorized

FDA Approves Tevimbra for the Treatment of Advanced or Metastatic Esophageal
Squamous Cell Carcinoma

BeiGene, Ltd., has announced that the U.S. Food and Drug Administration (FDA) has approved Tevimbra (tislelizumab-jsgr) as monotherapy for the treatment of adult patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.

Approval is based on the RATIONALE 302 trial, which met its primary endpoint in the intention-to-treat (ITT) population with a statistically significant and clinically meaningful survival benefit for Tevimbra compared with chemotherapy, highlighting its potential as an important treatment option for these patients.

Tevimbra is an IV injection and will be available in the U.S. in the second half of 2024.


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Poor Trial Results May Prompt Maker to Pull ALS Drug Relyvrio From Market

by News

Courtesy of HealthDay, by Robin Foster, HealthDay Reporter; Medically reviewed by Carmen Pope, BPharm

Following disappointing trial results, the maker of a controversial ALS drug may pull the medication off the market.

In a statement issued Friday, Amylyx Pharmaceuticals said that Relyvrio failed to help patients in a large follow-up study, but the company stopped just short of saying it will definitely pull the drug from the market. The drug is sold as Albrioza in Canada.

“Amylyx intends to share plans for Relyvrio/Albrioza in ALS, which may include voluntarily withdrawing Relyvrio/Albrioza from the market,” the company said in its statement. “At this time, Relyvrio/Albrioza and its related patient support program will continue to be available for people living with ALS. Amylyx has voluntarily decided to pause promotion of the medication during this time.”

Executives added that they were “surprised and disappointed” by the results and would announce their plans for the drug in the next two months.

Relyvrio was first approved by the U.S. Food and Drug Administration (FDA) in September 2022, following a lengthy, impassioned campaign by patients with ALS, a fatal muscle-wasting disease.

Unfortunately, the latest company study showed the drug did not slow the disease compared with a placebo treatment, and it also did not produce improvements on any secondary measures, such as muscle strength.

ALS is a devastating neurological disease that slowly destroys nerve cells and connections that are needed to walk, talk, speak, and breathe. Most patients die within three to five years of their diagnosis.

Relyvrio’s approval was mainly based on results from one small study that was criticized by some of the FDA’s scientists. An outside committee of experts also voted against the drug initially, before being swayed to back it at a follow-up meeting requested by patients. At the time, Amylyx noted it was continuing a larger follow-up study of more than 600 patients that would provide further data on the drug.

In a highly unusual move, Amylyx executives at that second advisory committee meeting told FDA regulators they would pull the drug from the market if follow up research didn’t confirm its efficacy. That commitment seemed to reassure FDA’s advisers, who then voted in favor of the drug’s approval, despite the questionable data.

Amylyx has also faced criticism for pricing the drug at $158,000 for a year’s supply, the Associated Press reported, and sales have been lackluster since Relyvrio’s launch in late 2022. Relyvrio combines two older drugs: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine.

Sources
• Amylyx Pharmaceuticals, news release, March 8, 2024
• Associated Press