Article Archives - Page 18 of 20

FDA approves treatment for rare Duchenne Muscular Dystrophy Mutation

The U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Exons are pieces of DNA that provide information for making proteins in a person’s genome). The agency approved Amondys 45 based on an increase in dystrophin (a protein that helps keep muscle cells intact) production in skeletal muscle observed in patients treated with the therapy. This is the first FDA-approved targeted treatment for patients with this type of mutation. Approximately 8% of patients with DMD have a mutation that is amenable to exon 45 skipping.

Dose: 30 mg/kg IV

Side effects: Upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.

Source: FDA

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Russia detects first case of H5N8 bird flu in humans

The H5N8 strain is deadly for birds, and this marks the first transmission of the strain from animals to humans and has reported the matter to the World Health Organization (WHO).

Humans and other mammals normally are not susceptible to infection by avian influenza A viruses. Nevertheless, several subtypes of avian influenza or bird-origin influenza viruses have infected humans; 3 of these subtypes have caused pandemics within the past century. At present, HPAI H5N1 is entirely an avian influenza subtype. Humans can become infected, but so far as is known, they must inhale or ingest massive viral doses from excreta or tissues of infected birds to do so.

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Zika vaccine candidate shows promise in Phase 1 Trial

The researchers randomized 100 healthy adult volunteers to an experimental Zika vaccine candidate known as Ad26.ZIKV.001 in either one-dose or two-dose regimens of 5×1010 viral particles (low dose) or 1×1011 viral particles (high dose) or placebo. Approximately half (55%) of the participants were women, and 72% were White.

Approximately 80% of patients in both two-dose groups showed antibody responses for a year after vaccination. Geometric mean titers (GMTs) reached a peak of 823.4 in the low-dose/low-dose group and 961.5 in the high-dose/high-dose group. On day 365, the GMTs for these groups were 68.7 and 87.0, respectively.

A single high-dose vaccine achieved a similar level of neutralizing antibody titers, but lower peak neutralizing responses than the two-dose strategies, the researchers noted.

Source: Medscape

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FDA: COVID-19 not transmitted by food or packaging

“After more than a year since the coronavirus disease 2019 (COVID-19) outbreak was declared a global health emergency, the U.S. Department of Agriculture, the U.S. Food and Drug Administration and the U.S. Centers for Disease Control and Prevention continue to underscore that there is no credible evidence of food or food packaging associated with or as a likely source of viral transmission of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the virus causing COVID-19,” said the statement attributed to Janet Woodcock, MD, acting commissioner of food and drugs at the U.S. Food and Drug Administration.

The statement noted that COVID-19 is a respiratory illness spread from person to person. Some researchers found small amounts of virus particles on food or food packaging, but the infection usually requires a much higher number of particles, the statement said.

Source: FDA

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Tocilizumab cuts deaths in hospitalized COVID-19 patients

Tocilizumab, sold under the brand name Actemra, is an intravenous anti-inflammatory monoclonal antibody drug used to treat rheumatoid arthritis. It was added to the trial in April 2020 for patients with COVID-19 who required oxygen and had evidence of inflammation.

The study data were from 2,022 COVID-19 patients who were randomly allocated to receive tocilizumab by intravenous infusion and who were compared with 2,094 patients randomly allocated to usual care alone. Researchers said 82% of all patients were taking a systemic steroid such as dexamethasone.

Results showed that treatment with tocilizumab significantly reduced deaths – with 596 (29%) of the patients in the tocilizumab group dying within 28 days, compared with 694 (33%) patients in the usual care group.

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FDA approves Evinacumab-dgnb for Homozygous familial hypercholesterolemia

The US FDA has approved the fully human monoclonal antibody evinacumab-dgnb (Evkeeza, Regeneron Pharmaceuticals) for use on top of other cholesterol-modifying meds in patients aged 12 and older with homozygous familial hypercholesterolemia (HoFH).

Mechanism: Evinacumab-dgnb is a fully-human monoclonal antibody that binds to and blocks the function of ANGPTL3.

Dose: 15 mg/kg) once a month via intravenous infusion.

Adverse Effects: Nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, pain in extremity and asthenia.

Cost: $450,000 per year

Extra notes: Reductions in LDL-C seen were observed as early as week 2 and maintained throughout the double-blind treatment period (week 24) and open-label trial period (through week 48).

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Pembrolizumab ‘Preferred Choice’ in MSI-H/dMMR Metastatic CRC

Pembrolizumab significantly improved progression-free survival compared with chemotherapy among patients with microsatellite instability–high/mismatch repair–deficient (MSI-H/dMMR) metastatic colorectal cancer, according to results from the KEYNOTE-177 study.

Study Details: The KEYNOTE-177 trial included 307 patients with confirmed, untreated MSI-H/dMMR metastatic colorectal cancer who were randomly assigned to receive pembrolizumab 200 mg every 3 weeks for up to 35 cycles (n = 153) or the investigators’ choice of chemotherapy (n = 154). Chemotherapy regimens included modified FOLFOX (5-fluorouracil, leucovorin, oxaliplatin) alone or in combination with either bevacizumab or cetuximab, or FOLFIRI (leucovorin, fluorouracil, irinotecan) alone or in combination with either bevacizumab or cetuximab. Patients in the chemotherapy group could cross over to pembrolizumab therapy after disease progression.

Source: Medscape

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Ruxolitinib as second line drug in Chronic GVHD

Ruxolitinib, a JAK inhibitor first marketed for use in myelofibrosis, is already approved for acute GVHD. The US Food and Drug Administration approved that indication last year on the basis of data from two previous trials, REACH 1 and REACH 2. The trials found that ruxolitinib was superior to best available therapy for treating patients with acute GVHD.

In the current REACH 3 study, Zeiser and colleagues compared ruxolitinib with best available therapy in 329 patients with moderate-to-severe cGVHD (both steroid dependent and steroid resistant).

Source: Medscape

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Human to human transmission of Chapare virus in Bolivia

Chapare hemorrhagic fever (CHHF) is a viral hemorrhagic fever caused by infection with Chapare virus. The Chapare virus is in the arenavirus family. Arenaviruses are usually spread to people through direct contact with infected rodents or indirectly through the urine or feces (droppings) of an infected rodent.

There have been two documented outbreaks of CHHF to date. The first occurred in 2003 in Chapare Province, Bolivia, which resulted in one fatal case. The second outbreak occurred in 2019 in Caranavi Province, Bolivia and resulted in five confirmed cases— three of which were fatal.

Transmission: Rodent reservoir of Chapare virus is unknown, similar arenaviruses are typically transmitted either through direct or indirect contact with the saliva, urine, and droppings of infected rodents.

Signs & Symptoms: The documented signs and symptoms of CHHF from the first and second outbreaks included some or all of the following:

fever
headache
joint and muscle pain
pain behind the eyes
stomach pain
vomiting
diarrhea
bleeding gums
rash
irritability

Diagnosis: real-time reverse transcription polymerase chain reaction (rRT-PCR). CDC advises a Biosafety Level 4 laboratory, strictly following all protocols for personal protection, sample inactivation and waste disposal.

Treatment: There is currently no treatment for CHHF. Supportive therapy is important for recovery from and survival of CHHF. This includes:

maintenance of hydration,
management of shock (eg, fluid resuscitation, administration of vasopressin stocks)
sedation
pain relief
transfusions (when necessary)

Source: CDC

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VEXAS Syndrome

Researchers from the National Institutes of Health (NIH) have discovered a new inflammatory disorder called Vacuoles, E1 enzyme, X-linked, Autoinflammatory and somatic Syndrome (VEXAS), which is caused by mutations in the UBA1 gene.

VEXAS causes symptoms that included blood clots in veins, recurrent fevers, pulmonary abnormalities and vacuoles (unusual cavity-like structures) in myeloid cells.