Monthly Archives: September 2022

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.

The U.S. Food and Drug Administration approved Regeneron/Sanofi’s Dupixent (dupilumab) for the treatment of adult patients with prurigo nodularis (PN). Dupixent is the first medication approved for PN, a rare inflammatory skin condition that can cause severe itching and have a significant negative impact on sleep and quality of life.

• Dupixent is an anti–interleukin (IL)-4 and anti–IL-13 biologic that was first approved to treat moderate to severe atopic dermatitis in 2017 and has since gained additional indications to treat several other type 2 inflammatory-driven conditions, such as asthma.

• In the Phase 3 PRIME and PRIME2 clinical trials, patients receiving Dupixent experienced superior itch reduction and achieved higher rates of clear or almost clear skin at 24 weeks compared with patients receiving placebo.

• IPD Analytics recommends prior authorization (PA) for Dupixent in the treatment of PN with a step through at least one medium- to super high-potency topical corticosteroid (TCS). Additionally, the PA criteria should match the labeled age and indication for PN. IPD also recommends including certain inclusion criteria from the PRIME and PRIME2 trials in the PA criteria.

• Utilization management of Dupixent should be high on payers’ radar, as utilization of the drug is expected to continue to grow for at least the next 5 years and biosimilar competition likely will not reach the market until 2031.

Celltrion USA announced that the U.S. Food and Drug Administration (FDA) has approved Vegzelma (bevacizumab-adcd), an anti-cancer monoclonal antibody treatment and a biosimilar to Avastin (bevacizumab), for the treatment of six types of cancer: metastatic colorectal cancer; recurrent or metastatic non-squamous non-small cell lung cancer (nsNSCLC); recurrent glioblastoma; metastatic renal cell carcinoma; persistent, recurrent, or metastatic cervical cancer; and epithelial ovarian, fallopian tube, or primary peritoneal cancer.

Vegzelma is a recombinant humanized monoclonal antibody which binds to vascular endothelial growth factor (VEGF), the key driver of vasculogenesis and angiogenesis, and thereby inhibits the binding of VEGF to its receptors Flt-1 (VEGFR-1), and kinase insert domain receptor (KDR) (VEGFR-2), on the surface of endothelial cells.

The FDA approval of Vegzelma was based on the totality of evidence, including the pivotal phase III trial in patients with metastatic or recurrent nsNSCLC. Results showed that as a first-line treatment, Vegzelma is highly similar to the reference product in terms of efficacy, safety and pharmacokinetics.

The FDA has approved chloroprocaine hydrochloride ophthalmic gel 3% (Iheezo; Harrow) for use as an ocular surface anesthesia. Iheezo is a sterile, single-use ophthalmic gel preparation administered by physicians. The treatment contains no preservatives and is safe and effective for ocular surface anesthesia, according to a Harrow press release.

• Iheezo is the first ophthalmic formulation of chloroprocaine hydrochloride, an anesthetic previously approved in injectable formulations under the brand names Nesacaine/Nesacaine- MPF for local anesthesia and Clorotekal for spinal anesthesia.
• This surface anesthesia is contraindicated in patients who have a history of hypersensitivity to any component of the medication. The drug should not be injected or administered intraoculary.
• Iheezo is physician-administered topically to the ocular surface prior to the planned surgical procedure. It is rapidly acting (about 1 to 5 minutes) and the anesthetic effect lasts around 20 minutes.
• The most common adverse reaction is mydriasis.
• Given the early FDA approval date, the commercial launch date for Iheezo will be slightly ahead of the original planned launch of May 2023.

Artivion, Inc., a leading cardiac and vascular surgery company focused on aortic disease, announced today that it has stopped the PROACT Xa clinical trial, a prospective, randomized, trial designed to determine if patients with an On-X mechanical aortic valve can be maintained safely and effectively on apixaban rather than on warfarin. The decision was based on the recommendation of the independent Data and Safety Monitoring Board (DSMB) of the trial due to lack of evidence supporting non-inferiority of apixaban to warfarin for valve thrombosis and thromboembolism.

• The PROACT Xa trial was designed to determine whether apixaban would yield equivalent safety to the standard anticoagulant, warfarin.
• The trial began enrolling in April 2020. The DSMB found that blood clots, resulting in stroke, occurred more frequently in patients receiving apixaban and that continuing the trial was unlikely to achieve the primary endpoint while possibly exposing patients to increased risk.

Adverse reactions or quality problems experienced with the use of a recalled product can be reported to

the FDA’s MedWatch Adverse Event Reporting Program online, by mail, or by fax.

• Submit a Report Online: https://www.accessdatfda.gov/scripts/medwatch/index.cfm
• Send a Report Via S. Mail or Fax:

Download a form from https://www.fda.gov/safety/reporting-serious-problems-fda/forms- reporting-fda or call 1-800-332-1088 to request a reporting form. Then complete and return to the address on the form or submit by fax to 1-800-FDA-0178.

Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.

*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.

Santen Inc., the U.S. subsidiary of Santen Pharmaceutical Co., Ltd. (Santen), and UBE Corporation (UBE) announced FDA approval of OMLONTI^® (omidenepag isopropyl ophthalmic solution) 0.002% eye drops.

• Omlonti is used for the reduction of elevated intraocular pressure (IOP) in patients with primary open-angle glaucoma or ocular hypertension.
• Omlonti is a relatively selective prostaglandin E2 (EP2) receptor agonist, indicated for the reduction of elevated intraocular pressure (IOP) and the recommended dosage is one drop in the affected eye(s) once daily in the evening.
• The risks identified with instillation of Omlonti include macular edema, including CME, in patients with aphakia, pseudophakia, or other risk factors for macular edema; ocular inflammation; the possibility of increased eye pigmentation; and eyelash changes.
• The product was released in five countries and regions beginning in February 2021.

The Food and Drug Administration granted accelerated approval to selpercatinib (Retevmo, Eli Lilly and Company) for adult patients with locally advanced or metastatic solid tumors with a rearranged during transfection (RET) gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options.

• Tumor types with responses included pancreatic adenocarcinoma, colorectal, salivary, unknown primary, breast, soft tissue sarcoma, bronchial carcinoid, ovarian, small intestine, and cholangiocarcinoma.
• The most common adverse reactions (≥25%) in patients were edema, diarrhea, fatigue, dry mouth, hypertension, abdominal pain, constipation, rash, nausea, and headache.
• The recommended selpercatinib dose is based on body weight.

Guerbet, a global medical imaging company, announced FDA approval of Elucirem (Gadopiclenol), a new macrocyclic GBCA for use in contrast-enhanced magnetic resonance imaging (MRI). The FDA is the first health authority to have approved Elucirem.

• Elucirem (Gadopiclenol) is a new macrocyclic gadolinium-based contrast agent with high relaxivity indicated for use in adults and children aged 2 years and older, for contrast-enhanced magnetic resonance imaging (MRI).
• The product is used to detect and visualize lesions with abnormal vascularity in the central nervous system (brain, spine, and associated tissues) and the body (head and neck, thorax, abdomen, pelvis, and musculoskeletal system).
• Elucirem will be marketed by Guerbet in the United States in bottle and pre-filled syringe form.