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The US Food and Drug Administration (FDA) has approved a new presentation of Menveo [Meningococcal (Groups A, C, Y, and W-135) Oligosaccharide Diptheria CRM197 Conjugate Vaccine] for individuals aged 10 to 55 years to help prevent invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, Y, and W.

The Menveo one-vial presentation now comes in a ready-to-use single vial giving healthcare providers a more convenient option. The Menveo one-vial presentation will initially be available to US federal customers, with broader availability anticipated in mid-2023.

Menveo (Meningococcal (Groups A, C, Y and W-135) Oligosaccharide Diphtheria CRM197 Conjugate Vaccine) is a vaccine indicated for active immunization to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroups A, C, Y and W-135.

The FDA amended the emergency use authorizations (EUAs) of the Moderna and Pfizer-BioNTech COVID-19 bivalent vaccines to authorize their use as a single booster dose in younger age groups.

Both vaccines target the Omicron BA.4 and BA.5 subvariants.

• The Moderna COVID-19 bivalent vaccine is authorized for administration as a single booster dose at least 2 months following completion of primary or booster vaccination in children 6 years of age and older.
• The Pfizer-BioNTech COVID-19 bivalent vaccine is authorized for administration as a single booster dose at least 2 months following completion of primary or booster vaccination in children 5 years of age and older.

FDA approves Krazati (adagrasib) for locally advanced or metastatic non-small cell lung cancer (NSCLC) with a KRASG12C mutation

The FDA has granted accelerated approval to Mirati Therapeutics’ Krazati (adagrasib), an oral KRAS G12C inhibitor, for the treatment of adult patients with KRAS G12C–mutated locally advanced or metastatic non–small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.

In 2022, it is estimated that approximately 236,740 adults in the United States will be diagnosed with lung cancer, and NSCLC is thought to account for 82% of those lung cancer diagnoses. An estimated 13%–14% of patients with NSCLC are thought to harbor the KRAS G12C mutation, representing approximately 20,000 to 25,000 people in the United States. According to Amgen, as many as 66% of patients with NSCLC present with advanced or metastatic disease at initial diagnosis.

Krazati has demonstrated a positive benefit-risk profile with accelerated approval based on the Phase 2 registration-enabling cohort of the KRYSTAL-1 study, evaluating Krazati 600 mg capsules administered orally twice daily in 116 patients with KRASG12C
-mutated advanced NSCLC who previously received treatment with a platinum-based regimen and an immune checkpoint inhibitor.

Also on December 12, 2022, the FDA approved Qiagen’s therascreen KRAS RGQ PCR kit and Agilent Technologies’ Resolution ctDx FIRST Assay as companion diagnostics for Krazati.

Krazati will directly compete with Amgen’s Lumakras (sotorasib), which was granted accelerated approval on May 28, 2021, for the same indication.

Mirati has priced Krazati at $240,000 per year; the estimated annual WAC for Lumakras is currently $231,000.

Read more…

The FDA has approved Furoscix (furosemide injection) a loop diuretic used for the at- home treatment of adults with congestion due to fluid overload in chronic heart failure.

scPharmaceuticals Inc., focused on developing and commercializing products that have the potential to optimize the delivery of infused therapies, advance patient care, and reduce healthcare costs, announced that the U.S. Food and Drug Administration (FDA) has approved Furoscix (furosemide injection), a proprietary formulation of furosemide delivered via an on-body infusor for the treatment of congestion due to fluid overload in adults with New York Heart Association Class II/III chronic heart failure.

Furoscix demonstrated 99.6% bioavailability and produced similar diuresis and natriuresis compared to intravenous furosemide. Furoscix is not indicated for emergency situations or in patients with acute pulmonary edema. The Furoscix infusion will last about 5 hours. Furoscix Infusor will deliver only an 80- mg dose. Furoscix is the first and only FDA-approved subcutaneous loop diuretic that delivers IV equivalent diuresis at home via the Furoscix Infusor.

The U.S. Food and Drug Administration on Friday allowed the use of GlaxoSmithKline’s (GSK.L) Boostrix vaccine during the third trimester of pregnancy to prevent whooping cough in infants younger than two months of age.

“When the Boostrix vaccine is given during pregnancy, it boosts antibodies in the mother, which are transferred to the developing baby,” the agency said.

While FDA’s approval of Boostrix has always included its use during pregnancy to protect the vaccinated individual, the latest decision expands its use to help prevent pertussis, commonly known as whooping cough, in infants younger than two months.

Alnylam has announced FDA approval of supplemental new drug application for Oxlumo (lumasiran) in advanced primary hyperoxaluria type 1.

The leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) approved a label expansion for Oxlumo^® (lumasiran), an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in pediatric and adult patients.

Oxlumo is administered once a month for three months, then monthly for patients weighing less than 10 kg, and quarterly for patients weighing more than 10 kg.

The most common adverse reaction (reported in ≥20% of patients) is injection site reactions.

Theratechnologies’ Trogarzo (ibalizumab-uiyk), an antiviral medicine that prevents human immunodeficiency virus (HIV) from multiplying in your body, has been approved by the FDA for 30-Second Intravenous (IV) Push, simplifying HIV treatment for heavily treatment-experienced population.

Trogarzo is used to treat the small percentage of patients who have multidrug-resistant human immunodeficiency virus type 1 HIV (MDR HIV-1 Infection) and who have failed other HIV therapies. In a clinical trial, most patients (33 of the 40 patients, or 83%) experienced a significant decrease in their HIV-RNA levels one week after Trogarzo was added to their ineffective antiretroviral regimens.

Trogarzo is administered intravenously (IV) once every 14 days by a trained medical professional

Catalyst Pharmaceuticals CPRX announced that the FDA approved its supplemental new drug application (sNDA) for Firdapse (amifampridine) tablets in 10 mg dosage to include pediatric patients (six years and older) for treating Lambert-Eaton myasthenic syndrome (“LEMS”).

The company submitted an sNDA to the FDA for the use of Firdapse in treating pediatric LEMS patients in the first quarter of 2022. LEMS is an ultra-rare disease autoimmune disorder characterized by muscle weakness of the limbs. Post the sNDA approval by the FDA, Firdapse is now a treatment option in the United States for all LEMS patients beyond six years of age.

Firdapse (amifampridine phosphate) is a nonspecific, voltage-dependent, potassium (K+) channel blocker for the treatment of Lambert Eaton myasthenic syndrome (LEMS) in adults and pediatric patients six years of age and older.

The FDA approved Relyvrio, a drug developed by Amylyx Pharmaceuticals, after extensive discussions about the safety and efficacy of the drug. Used to treat adults with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease, the drug was initially rejected by an FDA advisory panel in March of this year. Earlier this month, the panel reconvened and reanalyzed, voting in favor of Relyvrio after being presented with additional data. The final ruling was delivered on September 29, 2022.

“FDA approval of Relyvrio is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx.

The U.S. Food and Drug Administration (FDA) granted accelerated approval to LYTGOBI (futibatinib) for the treatment of adult patients with previously treated, bile duct cancer that has spread or cannot be removed by surgery. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

The approved recommended dosage of LYTGOBI is 20 mg orally (five 4 mg tablets) once daily until disease progression or unacceptable toxicity occurs. It is used in patients who have already received a previous treatment, and whose tumor has a certain type of abnormal FGFR2 gene. The tablets can be taken with or without food and swallowed whole, at approximately the same time each day.

Additional information regarding dosage and administration as well as warnings and precautions about ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity can be found in the full prescribing information.