Monthly Archives: February 2023

Reata Pharmaceuticals, Inc., a biopharmaceutical company focused on developing and commercializing novel therapies for patients with severe diseases, has received approval from the U.S. Food and Drug Administration for Skyclarys™ (omaveloxolone) as a treatment for Friedreich’s ataxia in adults and adolescents aged 16 years and older.

Friedreich’s ataxia is an ultra-rare, inherited neurodegenerative disorder that is typically diagnosed during adolescence and affects approximately 5,000 patients in the U.S. With this approval, the FDA granted a rare pediatric disease priority review voucher. The WAC Cost for a one-month supply is noted at $370,000.

Skyclarys™, an oral once-daily medication that has received Orphan Drug designation, is also under review in Europe by the European Medicines Agency (EMA).

Treatment with Skyclarys can cause an elevation in hepatic transaminases (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]. Skyclarys can cause changes in cholesterol and increases in B-type natriuretic peptide (BNP), a marker of cardiac function.

Women are advised not to breastfeed during treatment with Skyclarys.

Anticipated commercial drug supply of Skyclarys will be available in the second quarter of 2023.

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Pharmedica USA LLC is voluntarily recalling two lots of Purely Soothing, 15% MSM Drops to the consumer level. This product is being recalled due to non-sterility.

Risk Statement: Use of contaminated eye drops can result in the risk of eye infections that could result in blindness.

To date, Pharmedica USA LLC has not received any reports of adverse events or illness related to this recalled product.

Product was distributed worldwide by Purely Soothing LLC via online e-commerce and trade shows (Ex. Amazon Marketplace, etc.).

The eye drop is used as an anti-inflammatory aimed to assist with symptoms of ocular irritation and/or swelling and is packaged in white, cylindrical HDPE bottles. The eye drops (LOT#: 2203PS01, 1 oz, UPC 7 31034 91379 9; and LOT#: 1808051, ½ oz, UPC 7 31034 91382 9) have eye dropper caps and white lids. The product can be identified by the labels below.

Pharmedica USA, LLC is advising customers to immediately stop using the product and return it to the place of purchase. Wholesalers and retailers should stop distributing/return to Pharmedica USA LLC immediately or confirm that the product has been disposed of with proper verification.

Consumers with questions regarding this recall can contact Pharmedica USA LLC by phone number at +1 (623) 698 – 1752 or e-mail address at osm@pharmedicausa.com Monday to Friday between the hours of 8:00 AM MST (AZ) and 5:00 PM MST (AZ).. Consumers should contact their physician or healthcare provider if they have experienced any problems that may be related to taking or using this drug product.

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Global Pharma Healthcare is voluntarily recalling Batch No. H29 of Artificial Eye Ointment, distributed by Delsam Pharma as an over-the-counter product, due to possible microbial contamination. Additionally, some product packaging is leaking or may otherwise be compromised.

Risk Statement: Use of contaminated eye ointment may cause adverse events, including infection in the eye that could lead to blindness. To date, Global Pharma Healthcare has not received any reports of adverse events related to this product.

Artificial Eye Ointment (mineral oil 15%, white petrolatum 83%, 3.5 grams / 1/8 oz.) is used as an eye lubricant and to relieve dryness of the eyes. The affected product is packaged in a white aluminum tube within a paper carton. The product can be identified by the photos provided below. The product was distributed nationwide in the United States, and by Delsam through internet retail sites. Delsam Pharma’s NDC for this product is 72570-122-35, and its UPC code is 3 72570 012235 3.

Global Pharma Healthcare is notifying the brand owner and importer of this product, Delsam Pharma, about this recall, and is requesting that wholesalers, retailers, and customers who have the recalled product should stop any use and discard the product safely and appropriately.

Consumers with questions regarding this recall can contact the distributor Delsam Pharma, LLC by phone at 1-866-826-1306 or by e-mail at delsampharma@yahoo.com from Monday to Friday from 11am to 4pm EST.

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Pfizer Inc. has announced that the U.S. Food and Drug Administration has granted Priority Review for the company’s Biologics License Application (BLA) for elranatamab, an investigational B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb), for the treatment of patients with relapsed or refractory multiple myeloma (RRMM). Priority Review is intended to direct attention and resources from regulatory authorities toward drugs that, if approved, could offer significant improvements over existing options for serious conditions in order to make these drugs available to patients faster. The FDA’s decision on the application is expected in 2023. The European Medicines Agency (EMA) has also accepted elranatamab’s marketing authorization application (MAA). The company is working closely with the EMA to facilitate their review and will provide updates on timing as appropriate.

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• • If approved, pozelimab would be the first and only treatment for those living with CHAPLE.
• • CHAPLE is an ultra-rare hereditary immune disease that causes overactivation of the complement system, leading to potentially life-threatening abdominal and cardiovascular symptoms.

Regeneron Pharmaceuticals, Inc. has announced the U.S. Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for pozelimab as a treatment for adults and children as young as 1 year of age with CHAPLE disease (also known as CD55 deficiency with Hyperactivation of complement, Angiopathic thrombosis and Protein Losing Enteropathy or CD55-deficient protein-losing enteropathy). There are currently no approved treatments for CHAPLE, an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. Pozelimab is an investigational fully-human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation. The target action date for the FDA decision is August 20, 2023.

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Travere Therapeutics, Inc. has announced that the U.S. Food and Drug Administration has granted accelerated approval to Filspari™ (sparsentan) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g.

This indication is granted under accelerated approval based on reduction in proteinuria. It has not been established whether Filspari slows kidney function decline in patients with IgAN. The continued approval of Filspari may be contingent upon confirmation of a clinical benefit in the ongoing Phase 3 PROTECT Study, which is designed to demonstrate whether Filspari slows kidney function decline.

Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in the fourth quarter of 2023 and are intended to support traditional approval of Filspari.

Filspari, a once-daily oral medication is designed to selectively target two critical pathways in the disease progression of IgAN (endothelin-1 and angiotensin II) and is the first and only non-immunosuppressive therapy approved for the treatment of this condition. IgAN is a rare kidney disease) and a leading cause of kidney failure due to glomerular disease, affecting up to 150,000 people in the U.S., with approximately 30,000 to 50,000 of such patients estimated to be addressable under the indication approved via accelerated approval.

• • Advise patients with symptoms suggesting hepatotoxicity to immediately stop treatment with
• Filspari and seek medical attention.
• • Monitor serum potassium periodically and treat appropriately.
• • Fluid retention may occur.
• • There was a greater incidence of hypotension-associated adverse events, some serious, in
• patients treated with Filspari compared to irbesartan.
• • Avoid use of Filspari in patients with any hepatic impairment.
• • Filspari can cause fetal harm.

The Company expects Filspari to be available beginning the week of February 27, 2023, and will be
providing a comprehensive patient support program throughout the patient’s treatment journey.

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The FDA has approved Altuviiio™ (Sanofi) [Antihemophilic Factor (Recombinant), FcVWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, highsustained factor VIII replacement therapy. This is the first approval of Altuviiio.

Altuviiio is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. It is the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis. Altuviiio has a 3- to 4-fold longer half-life relative to standard and extended half-life factor VIII products.

Hemophilia A is a rare, lifelong condition in which the ability of a person’s blood to clot properly is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and potentially impact quality of life.

The FDA evaluated the application under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Regulatory submission in the EU is anticipated in the second half of 2023. The European Commission granted Orphan Drug designation in June 2019.

To ensure that patients have access to the improved bleed protection provided by Altuviiio, Sanofi will
price Altuviiio at parity to the annual cost of treating a prophylaxis patient on Eloctate® [Antihemophilic
Factor (Recombinant), Fc Fusion Protein].

Sobi and Sanofi have collaborated on the development and commercialization of Altuviiio™ in the US,
where it is expected to be commercially available in April.

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Chiesi Global Rare Diseases, (a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group, Chiesi Group), has announced that the FDA has approved Lamzede (velmanase alfa-tycv) the first and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients.

AM is an ultra-rare, progressive lysosomal storage disorder caused by deficiency in the enzyme αmannosidase, and Lamzede is indicated for the treatment of both adult and pediatric patients.

The prevalence of AM is approximately one in every 500,000 to one in every 1,000,000 babies born worldwide. AM results in the body’s cells being unable to properly break down certain groups of complex sugars. The buildup of sugars can affect many of the body’s organs and systems. Effects of the disease vary significantly from person to person and progress over time.

The most common adverse reactions (incidence >20%) are hypersensitivity reactions including
anaphylaxis, nasopharyngitis, pyrexia, headache, and arthralgia.

Lamzede may cause embryo-fetal harm when administered to a pregnant female. A negative pregnancy status must be verified in females of reproductive potential prior to initiating the medication. Patient should be advised to use effective contraception during treatment and for 14 days after the last dose of Lamzede.

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IBSA Pharma Inc. Issues Voluntary Nationwide Recall of Select Lots of TIROSINT®-SOL (levothyroxine sodium) Oral Solution Due to Subpotency

IBSA Pharma Inc. is voluntarily recalling 27 lots of TIROSINT®-SOL (levothyroxine sodium) Oral Solution to the consumer level. This voluntary recall has been initiated because these lots may be subpotent. The company’s analyses show a slight decrease below 95.0% of its labeled amount in levothyroxine sodium (T4) for some lots.

This recall does not apply to TIROSINT® (levothyroxine sodium) capsules.

Risk Statement: Patients being treated for hypothyroidism (underactive thyroid), who receive subpotent TIROSINT®-SOL, may experience signs and symptoms of hypothyroidism (underactive thyroid) which may
include, fatigue, increased sensitivity to cold, constipation, dry skin, puffy face, hair loss, slow heart rate, depression, swelling of the thyroid gland and/or unexplained weight gain or difficulty losing weight. Over or under-treatment with TIROSINT® SOL may have negative effects on growth and development, cardiovascular function, bone metabolism, reproductive function, cognitive function, emotional state, gastrointestinal function, and glucose and lipid metabolism.

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Shop Me Ca of Herndon, VA is recalling its 10-gram tubes of “Diep Bao Cream” because they have the potential to be contaminated with lead. People with high blood levels of lead may show no symptoms, but the condition may cause damage to the nervous system and internal organs. Acute lead poisoning may cause a wide range of symptoms, including abdominal pain, muscle weakness, nausea, vomiting, diarrhea, weight loss, and bloody or decreased urinary output. Children are particularly vulnerable to lead poisoning. If a child is exposed to enough lead for a protracted period of time (e.g., weeks to months) permanent damage to the central nervous system can occur. This can result in learning disorders, developmental defects, and other long-term health problems.

Diep Bao Cream was sold nationwide through Shop Me Ca’s Facebook page and a Vietnamese Moms’ Facebook groups: Hội Mẹ Việt Nuôi con Tại Mỹ, Mẹ Việt Tại Mỹ, Chuyên Đồ Bầu và Chăm Sóc Sau Sinh, and Nguyễn Ngọc. These Facebook groups are not associated with Shop Me Ca.