Article Archives - Page 5 of 21

FDA approves Setmelanotide for obesity in Bardet-Biedl Syndrome

The US FDA has approved Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, is the first FDA-approved therapy for BBS, a rare genetic disorder that impairs a hunger signal along the melanocortin-4 receptor (MC4R) pathway.

Individuals with Bardet-Biedl Syndrome typically have obesity that starts at age 1 along with insatiable hunger (hyperphagia). Available weight management options are generally unsuccessful. Other symptoms may include retinal degeneration, reduced kidney function, or extra digits of the hands or feet.

The most common adverse reactions (with an incidence ≥ 20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.

Source: Medscape

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Massive breakthrough as rectal cancer disappears in every patient in drug trial

Patients with locally advanced rectal cancer and tumors with deficient mismatch repair (dMMR) have shown a remarkable response to treatment with the programmed cell death-1 (PD-1) inhibitor dostarlimab (Jemperli).

So far, the study has involved only 12 patients, but all of them have had a clinical complete response to treatment. They continue to show no signs of cancer (during follow-up ranging from 6 to 25 months) and have not undergone surgery or had radiation and chemotherapy, which are the standard treatment approaches.

Dostarlimab is already approved by the US Food and Drug Administration for use in the treatment of recurrent or advanced endometrial cancer with dMMR. Rectal cancer is an off-label use.

All patients had mismatch repair-deficient stage 2 or 3 rectal adenocarcinoma. The authors note that these tumors respond poorly to standard chemotherapy regimens, including neoadjuvant chemotherapy. The median age of enrolled patients was 54 years and 62% were women.

Source: NEJM

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IN.PACT 018 Drug-Coated Balloon approved for PAD

The US FDA has approved the IN.PACT 018 drug-coated balloon (DCB) for the treatment of peripheral arterial disease.

The paclitaxel-coated balloon is indicated for percutaneous transluminal angioplasty of de novo, restenotic, or in-stent restenotic lesions up to 360 mm in length with vessel diameters of 4 to 7 mm, located in the superficial femoral and popliteal arteries.

The IN.PACT 018 DCB is built on the same technology as the IN.PACT Admiral DCB (0.035 inch guidewire compatible), but is engineered to cross tight lesions, provide better deliverability, and is 0.018 inch guidewire compatible.

Source: Medscape

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First Line for Esophageal Cancer is now Immunotherapy

Immunotherapy with nivolumab (Opdivo) is now approved in the United States for first-line use in the treatment of unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).

The new approval for the drug, a programmed cell death ligand-1 (PD-L1) inhibitor, is for use in this patient population regardless of PD-L1 status. The indication also specifies that nivolumab is to be used together with chemotherapy (with a fluoropyrimidine- and platinum-containing regimen) or in combination with ipilimumab (Yervoy), an immunotherapy with a different mechanism of action.

Source: Medscape

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Rare monkeypox detected in UK: Check symptoms, treatment & other details

A patient was found to be infected with monkeypox virus in the United Kingdom, who traveled to the country from Nigeria.

Monkeypox is caused by monkeypox virus, a member of the Orthopoxvirus genus in the family Poxviridae. Monkeypox is a viral zoonotic disease that occurs primarily in tropical rainforest areas of Central and West Africa and is occasionally exported to other regions.

The incubation period (interval from infection to onset of symptoms) of monkeypox is usually from 6 to 13 days but can range from 5 to 21 days.

Monkeypox typically presents clinically with fever, rash and swollen lymph nodes.

Monkeypox virus is mostly transmitted to people from wild animals such as rodents and primates, but human-to-human transmission also occurs.
Monkeypox virus is transmitted from one person to another by contact with lesions, body fluids, respiratory droplets and contaminated materials such as bedding.

The clinical presentation of monkeypox resembles that of smallpox, a related orthopoxvirus infection which was declared eradicated worldwide in 1980.

Vaccinia vaccine used during the smallpox eradication programme was also protective against monkeypox. A new third generation vaccinia vaccine has now been approved for prevention of smallpox and monkeypox. Antiviral agents are also being developed.

Source: WHO

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Saliva test for diagnosing endometriosis

The prospective ENDO-miRNA study included saliva samples obtained from women with chronic pelvic pain suggestive of endometriosis. Exploratory procedures were performed to look for lesions. All the patients underwent either a laparoscopic procedure (therapeutic or diagnostic laparoscopy) and/or MRI imaging. For the patients who underwent laparoscopy, diagnosis was confirmed by histology. For the patients diagnosed with endometriosis without laparoscopic evaluation, all had MRI imaging with features of deep endometriosis.

The respective sensitivity & specificity for the diagnostic miRNA signature were 96.7%, 100% respectively.

The study’s results support the use of a saliva-based miRNA signature for diagnosing whether a patient is discordant/complex (chronic pelvic pain suggestive of endometriosis and both negative clinical examination and imaging findings) or has early-stage or advanced-stage endometriosis.

Source: Medscape

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FDA approves new immunotherapy for metastatic melanoma

The FDA approved a combination nivolumab/relatlimab-rmbw immune checkpoint inhibitor (Opdualag) for unresectable or metastatic melanoma in adults and children 12 years or older.

Dose: 480 mg nivolumab + 160 mg relatlimab IV every 4 weeks.

Adverse effects: Musculoskeletal pain, fatigue, rash, pruritus, and diarrhea were the most common adverse reactions with combination nivolumab/relatlimab, occurring in 20% or more of RELATIVITY-047 trial participants. Adrenal insufficiency, anemia, colitis, pneumonia, and myocardial infarction were the most frequent serious adverse reactions, but each occurred in less than 2% of patients. There were three fatal adverse events in the trial due to hemophagocytic lymphohistiocytosis, acute lung edema, and pneumonitis.

Source: BMS

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FDA approves Anifrolumab as first new Lupus treatment in more than 10 years

Anifrolumab, an inhibitor of type 1 interferons, received approval from the Food and Drug Administration for the treatment of adults with moderate to severe systemic lupus erythematosus (SLE) who are receiving standard therapy.

Dose: 300 mg concentrate for solution for infusion.

Adverse Effects: The most common adverse reactions are upper respiratory tract infection, bronchitis, infusion-related reaction, and herpes zoster. The most common serious adverse reaction was herpes zoster.

Source: FDA

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First drug for cold agglutinin disease

The US FDA approves first drug for Cold Agglutinin Disease. Sutimlimab-jome (Brand name: ENJAYMO) is a classical complement inhibitor indicated to decrease the need for red blood cell (RBC) transfusion due to hemolysis in adults with cold agglutinin disease.

Dose: Injection: 1,100 mg/22 mL (50 mg/mL) in a single-dose vial

Adverse effects: Most common adverse reactions (incidence ≥10%) are respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and
peripheral edema.

Source: FDA

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First treatment for rare Uveal Melanoma

The drug is tebentafusp-tebn (Kimmtrak, from Immunocore Limited), which acts as a bispecific gp100 peptide-HLA-directed CD3 T-cell engager. It is the first of a novel class of T-cell receptor (TCR) bispecific immunotherapies under development at the company.

It is also the first and only therapy for the treatment of unresectable or metastatic uveal melanoma to be approved by the FDA, and it is the first therapy to demonstrate a survival benefit to patients with this disease. However, the indication specifies that the drug is for use only in patients who are HLA-A*02:01-positive.

Source: Immunocore