Inborn Errors Of Metabolism
The safety and efficacy of Nityr have been established based on studies of another oral formulation of nitisinone in patients with HT-1.
Today's FDA approval follows the Agency's recent approval for 23 other residual function mutations based on in vitro data.
After adjustment for age, household income, and residential area, the multivariable-adjusted odds ratios for MetS for the upper stratum were 5.79 and 6.20 in boys and girls, respectively.
The Orphan Drug designation was supported by data from preliminary studies showing that ALLN-177 significantly decreased urinary and plasma oxalate in several rodent and porcine models.
According to the Company, SOBI003 is currently in the late pre-clinical phase and its first clinical trial is expected to commence in 2018.
The investigational drug is intended to substitute the deficient PAH enzyme with a PEGylated phenylalanine lyase enzyme to break down Phe.
Synthetic Biotic medicines utilize synthetic biology to reprogram probiotic bacteria to perform critical functions that compensate for those missing or damaged due to a particular disease.
The FDA's decision was based on analyses of in vitro data and real-world clinical data spanning over five years on the safety and efficacy of Kalydeco.
There is currently no approved treatment for lgG4-RD, which is a newly recognized disorder and is estimated to affect 40,000 individuals in the U.S.
However the authors emphasized that the enzyme was not tested and is not recommended for use in patients with celiac disease, as even a small amount of gluten can inflict long-term damage in these patients.
The FDA is recommending that clinicians prioritize access to the unapproved lot of Sucraid for patients with severe CSID and for patients with evidence of malnutrition.
The researchers found that of the 72 subjects taking testosterone, at each visit significant increases in hemoglobin/hematocrit levels and BMI were recorded, as was a decrease in high-density lipoprotein level.
Results showed that the ivacaftor/tezacaftor group reached the primary endpoint with a mean absolute improvement in ppFEV1 of 4% points from baseline vs. placebo (P<0.0001).
While the GHR tests may provide genetic risk information, they cannot determine a person's overall risk for developing a disease, as other factors beyond genetics (ie, environment, lifestyle) may also influence risk.
The researchers found that new antibiotic treatment was associated with an increased likelihood of recovery to 90% of baseline (P<0.001), especially for hospitalization compared to no new antibiotic.
The FDA has accepted and granted Priority Review to the New Drug Application (NDA) of Ryanodex (dantrolene sodium; Eagle) for the treatment of exertional heat stroke (EHS).
The FDA has approved the supplemental new drug applicationfor Orfadin (nitisinone capsules; Sobi) which allows for an extension of shelf life for the 20mg capsules from 24 months to 36 months.
The FDA has granted Orphan Drug designation to Sangamo's genome editing product candidate, SB-913, for the treatment of Mucopolysaccharidosis Type II (MPS II).
The Food and Drug Administration has approved the marketing of the Seeker System (Baebies, Inc.) for the screening of four rare lysosomal storage disorders (LSDs) in newborns.
Eagle announced the complete submission to the Food and Drug Administration (FDA) of its New Drug Application (NDA) for Ryanodex for the treatment of exertional heat stroke (EHS).
The Food and Drug Administration (FDA) has granted Orphan Drug designation for Concert Pharmaceuticals' CTP-656 (deuterated ivacaftor) for the treatment of cystic fibrosis.
New research indicates that patients with metabolic syndrome (MetS) may need significantly more vitamin E than healthy patients, and that conventional blood tests may not provide an accurate measure of vitamin E levels.
PhaseRx announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation for PRX-OTC for the treatment of ornithine transcarbamylase deficiency (OTCD).
Ledipasvir and sofosbuvir yielded 100% SVR in patients with thalassemia major, a genetic disease, who become infected with hepatitis C virus genotypes 1 and 4.
Eloxx announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to ELX-02, its propriety drug candidate for the treatment of mucopolysaccharidosis type 1 (MPS I).
New research has found that antenatal B12 deficiency may be tied to higher than normal leptin levels in offspring, which can lead to type 2 diabetes.