Monthly News Roundup - May 2024
FDA Clears GSK’s Benlysta Autoinjector for Systemic Lupus Erythematosus (SLE) in Children
In May, the U.S Food and Drug Administration (FDA) cleared GSK’s Benlysta subcutaneous autoinjector (given as an injection under the skin) to be used in children 5 years and older for the treatment of systemic lupus erythematosus (SLE).
- Lupus is a chronic, multi-organ (systemic) inflammatory disease that occurs when the immune system attacks its own tissues. SLE is the most common form of lupus. Lupus nephritis is a serious inflammation of the kidneys which can lead to dialysis or a kidney transplant.
- Benlysta (belimumab) was first approved in March 2011. It’s a B-lymphocyte stimulator (BLyS)-specific inhibitor approved for active systemic lupus erythematosus (SLE) and active lupus nephritis in adults and children 5 years and older who are receiving other lupus medicines.
- This approval will allow children to be treated at home after caregiver training. Previously, children with SLE could only receive this treatment as a one-hour intravenous (into a vein) infusion by a healthcare provider in a hospital or clinic setting. The subcutaneous dosage form is not yet approved for use in children with lupus nephritis.
- Benlysta subcutaneous dosage for children with SLE is weight-based. Patients weighing greater than or equal to 40 kg (88 lb) receive 200 mg once weekly, and those weighing 15 kg (33 lb) to less than 40 kg receive 200 mg once every 2 weeks.
- The most common side effects with Benlysta include: nausea, diarrhea, fever, common cold symptoms, bronchitis (persistent cough), insomnia (trouble sleeping), pain in extremity (like your arm or leg), depression, migraine headache, injection site reactions (with subcutaneous injection), and pharyngitis (sore throat).
- GSK has stated the 200 mg Benlysta autoinjector will be available immediately for caregivers of children 5 years of age and older with SLE who are receiving standard therapy.
CAR-T Cell Therapy Breyanzi Shows Rapid Response for Follicular Lymphoma
In May, the FDA granted accelerated approval to Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) for the treatment of adults with relapsed or refractory follicular lymphoma (lymphoma that has returned or not responded to other treatments). Breyanzi is a CD-19 directed chimeric antigen receptor (CAR) T cell therapy given as a single intravenous (IV) infusion in a REMS-certified healthcare facility.
- Follicular lymphoma is a type of low grade non-Hodgkin lymphoma blood cancer where too many lymphocytes (a type of white blood cell) aggregate in the lymph nodes or organs. Symptoms may include swollen lymph nodes, fever, stomach or chest pain.
- Accelerated approval was based on the Phase 2 TRANSCEND FL study in 94 patients, demonstrating an overall response rate (ORR) of 95.7% (95% CI: 89.5-98.8). ORR is defined as the percentage of patients achieving a partial or complete response (CR) per Lugano criteria. CR rate was 73.4% (95% CI: 63.3-82) with a median time to response of one month (range: 0.6-3.3) and a median duration of response (DOR) not reached (95% CI: 18.04-NR), with 80.9% of responders remaining in response at 12 months, and 77.1% of responders remaining in response at 18 months.
- Continued approval for follicular lymphoma may depend upon verification of clinical benefit in further clinical studies.
- Breyanzi carries a Boxed Warning, the FDA’s most stringent safety warning, detailing Cytokine Release Syndrome (CRS) and nervous system toxicities which can be fatal or life-threatening, and the risk of secondary blood cancers. It is available only through the restricted Breyanzi REMS program to help ensure safe use.
- Serious adverse effects can include allergic reactions, infections, prolonged low blood cell counts, low antibody production and risk of secondary cancers. Common side effects (≥30%) in patients with follicular lymphoma include cytokine release syndrome and laboratory changes like decreased white blood cell counts.
- Breyanzi is also labeled to treat adult patients with relapsed or refractory large B-cell lymphoma, and chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Opuviz and Yesafili Both Approved as Interchangeable Biosimilars to Eylea
In May, the FDA cleared Opuviz (aflibercept-yszy) and Yesafili (aflibercept-jbvf), the first interchangeable, biosimilar products to Eylea, an intravitreal injection used to treat retinal eye diseases.
- Both Opuviz and Yesafili are approved to treat neovascular (wet) age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema and diabetic retinopathy. They are administered by intravitreal injection (in the back of a numbed eye), usually every 4 to 8 weeks.
- Unlike Eylea, the new interchangeable biosimilars are not approved for retinopathy of prematurity.
- An interchangeable biosimilar is a biologic that can be automatically substituted by a pharmacist for the reference product (in this case, Eylea), depending upon state law, without contacting the prescriber. It also means the reference biologic and the new biosimilar can be switched back and forth without a risk of changes in safety or effectiveness. Use of biosimilars are meant to generate cost savings for both the patient and the healthcare system.
- Approval of Opuviz and approval of Yesafili were supported by a comprehensive review of scientific evidence demonstrating they are highly similar to Eylea with no clinically meaningful differences.
- Warnings and precautions for both include endophthalmitis (an inflammation and possible infection of the inner eye), retinal detachments, and retinal vasculitis (eye / blood vessel inflammation) with or without occlusion, increases in intraocular pressure (pressure inside your eyes), and risk of arterial thromboembolic (clotting) events.
- Common adverse reactions include eye bleeding, eye pain, cataracts, vitreous detachment (separation of gel-like fluid from retina in eye), vitreous floaters, and increased intraocular pressure.
- Opuviz is manufactured by Samsung Bioepis and Yesafili is made by Biocon Biologics Inc.
FDA Clears Amgen’s Bkemv: An Interchangeable Biosimilar to Soliris
Amgen’s Bkemv (eculizumab-aeeb) is now approved as the first interchangeable biosimilar to Soliris (eculizumab). Bkemv is indicated for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, two rare diseases characterized by the breakdown of red blood cells.
- Bkemv is not labeled to treat myasthenia gravis or neuromyelitis optica spectrum disorder (NMOSD) in adults, two additional approved uses for Soliris.
- An interchangeable biosimilar can automatically be substituted for the reference product (in this case, Soliris) by a pharmacist, depending upon state laws. The pharmacist will not need to contact the prescriber to get an approval. It also means the reference biologic and the new biosimilar can be switched back and forth in a patient without a risk of changes in safety or effectiveness.
- Interchangeable products are meant to be more affordable, more likely to be covered by insurance and make it easier for patients to access a biologic medicine.
- Bkemv is a monoclonal antibody and works by binding to the complement C5 protein to inhibit activation of the complement system, a part of the body’s immune system.
- Bkemv carries a Boxed Warning for an increased risk for meningococcal infections, which may be fatal. Due to serious safety risks, Bkemv is available only through a restricted program called the Bkemv Risk Evaluation and Mitigation Strategy (REMS).
- Bkemv has the same safety warnings and is expected to have the same adverse reactions as Soliris for the approved indications, including headache, nasopharyngitis (common cold), back pain, nausea, diarrhea, high blood pressure, and upper respiratory infection, among others.
Myhibbin: A Ready-to-Use Oral Suspension to Prevent Organ Rejection
In May the FDA approved oral Myhibbin (mycophenolate mofetil), an antimetabolite immunosuppressant used in combination with other immunosuppressants to prevent organ rejection in patients who are at least 3 months of age who received a liver, heart, or kidney transplant.
- Myhibbin contains mycophenolate mofetil which was first approved in 1995 under the brand name CellCept. Myhibbin is a liquid formulation of mycophenolate mofetil (200 mg/mL) that does not require reconstitution and is the first ready-to-use oral liquid formulation of mycophenolate.
- Myhibbin is taken as an oral suspension twice a day. Your healthcare provider will determine your exact dose.
- Myhibbin works by weakening your body's immune system to lower the chance of "rejecting" a transplanted organ. Organ rejection can occur when the immune system treats the transplanted organ as foreign and attacks it.
- Myhibbin carries a Boxed Warning for embryofetal toxicity (first trimester pregnancy loss, congenital malformations), malignancies (for example: lymphoma, skin cancers), and serious bacterial, viral, fungal and protozoal infections. Warnings and precautions include blood dyscrasias (blood disorder), gastrointestinal (stomach area) complications, hypoxanthine-guanine phosphoribosyl-transferase (enzyme) deficiency (may lead to overproduction and accumulation of uric acid and gout symptoms) and acute inflammatory syndrome.
- Common side effects include diarrhea, leukopenia (low white blood cells), infections and vomiting. Myhibbin may also affect the ability to drive or operate machinery.
- Myhibbin is manufactured by Azurity Pharmaceuticals, Inc. It’s expected to become commercially available in U.S. pharmacies in the second quarter of 2024.
First-in-Class Imdelltra Granted Accelerated Approval for Aggressive Small Cell Lung Cancer
This past month the FDA approved Amgen’s first-in-class Imdelltra (tarlatamab-dlle) for the treatment of adults with extensive stage small cell lung cancer (ES-SCLC) whose disease has worsened after treatment with platinum-based chemotherapy (treatment either did not work or is no longer working).
- Extensive-stage small cell lung cancer (ES-SCLC) is cancer that has spread in the body: to the other lung, to lymph nodes in the chest, or to other areas. About 70% of people diagnosed with small cell lung cancer have ES-SCLC. Less than 3% of patients with ES-SCLC live longer than five years.
- Imdelltra works by activating the patient's own T cells to attack DLL3-expressing tumor cells. Imdelltra is a bispecific delta-like ligand 3 (DLL3)-directed CD3 T cell engager (a Bispecific T-cell engager or BiTE). Bispecific means it works at two different targets - at the DLL3 expressed on the tumor cell and at CD3 protein on T-cells. Imdelltra helps to bring these two targets together to activate the immune system to attack the cancer.
- Approval was based on data from the Phase 2 DeLLphi-301 study in patients with SCLC who had failed two or more prior lines of treatment, and had received the 10 mg every 2 weeks dosing (Q2W) regimen in 99 patients. Results demonstrated an objective response rate (ORR) of 40% (95% CI: 31, 51) and median duration of response (DoR) of 9.7 months (CI: 2.7, 20.7+). The median overall survival (mOS) was 14.3 months, but final survival data is forthcoming. Continued approval of Imdellyra may depend upon continued benefits seen in additional clinical trials.
- Imdelltra carries a Boxed Warning for serious or possibly fatal side effects like Cytokine Release Syndrome (CRS) and nervous system (neurologic) problems. Other serious side effects include low blood cell counts (cytopenias), infections, liver toxicity, hypersensitivity (allergic) reactions, neurological problems, and harm to an unborn baby.
- The most common side effects (in at least 20% of patients) are: Cytokine Release Syndrome (CRS), fatigue and tiredness, fever, metallic taste in mouth, decreased appetite, muscle / bone pain, constipation, nausea. Your healthcare provider will monitor you closely for side effects.
- Imdelltra is administered as an intravenous infusion (through a needle placed in a vein) over 1-hour. Due to the risk of CRS, patients will receive Imdelltra utilizing a “step-up dosing schedule”. Patients need to remain in an appropriate healthcare setting for initial infusions to be monitored.
mRESVIA becomes the third RSV vaccine to be approved
The approval of mRESVIA from Moderna rounds up this month's new drug approvals. mRESVIA is approved for adults aged 60 years and older to immunize them against lower respiratory tract disease (LRTD) caused by the respiratory syncytial virus (RSV).
- RSV is a common virus that affects the lungs and breathing passages. It can cause severe disease (pneumonia, hospitalization, and death) in young infants and older adults due to age-related declining immunity or underlying conditions. The CDC recommends the respiratory syncytial virus vaccine (RSV vaccine) vaccine for infants, toddlers, and adults 60 years and older as they are most at risk of getting very sick with RSV.
- mRESVIA is a modified RNA vaccine that encodes for the RSV F glycoprotein which induces an immune response and the formation of antibodies against RSV pre-F protein which protects against severe symptoms of LRTD caused by RSV.
- mRESVIA is the third vaccine to be approved for RSV, and the only modified RNA vaccine. The other 2 RSV vaccines are Arexvy (approved for pregnant women and adults aged 60 and older) and Abrysvo (only approved for adults aged 60 and older). The most common side effects are injection site pain, fatigue/tiredness, headache, muscle pain, joint pain, underarm swelling or tenderness, and chills.
- It is given as an intramuscular injection, usually into the deltoid muscle of the upper arm.
Posted May 2024
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