Category: FDA Approvals

Novel Kinase Inhibitor Combination Approved for Advanced Melanoma

Continue reading “Novel Kinase Inhibitor Combination Approved for Advanced Melanoma”

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Moxidectin, First New Treatment for River Blindness in 30 Years, Approved by FDA

The US FDA has approved the antihelminthic drug moxidectin (developed by Medicines Development for Global Health) for the treatment of onchocerciasis.¹

Merck’s ivermectin, developed in the late 1970s, is the only other pharmaceutical agent approved for treating onchocerciasis.  Moxidectin has outperformed ivermectin in clinical trials, demonstrating superior efficacy and comparable safety. Continue reading “Moxidectin, First New Treatment for River Blindness in 30 Years, Approved by FDA”

Arthritis Drug Tofacitinib Approved for Severe Ulcerative Colitis | FDA

The US FDA has approved tofacitinib (Xeljanz, Pfizer) for the treatment of moderate-severe ulcerative colitis (UC).

Tofacitinib

  • Janus kinase inhibitor (blocks STAT transcription factor activation and inflammatory signaling)
  • First oral agent approved for chronic use in UC
  • 10 mg tofacitinib PO BID induced remission in 17-18% of patients (vs. 8.2% placebo) after 8 weeks
  • Of patients that responded after 8 week trial, 40.6% who continued 10-mg tofacitinib for 52 weeks achieved sustained remission (versus 11.1% placebo)
  • Adverse events (common):  arthralgia, diarrhea, elevated cholesterol, headache, shingles, rash, and upper respiratory infection
  • Adverse events (severe): malignancy (e.g., lymphoma), serious opportunistic infections (FDA black-box warnings)

Continue reading “Arthritis Drug Tofacitinib Approved for Severe Ulcerative Colitis | FDA”

Novel Enzyme Therapy Approved for Genetic Disease Phenylketonuria | FDA

The US FDA has approved pegvaliase (Palynziq®, BioMarin Inc) for the treatment of phenylketonurea (PKU) uncontrolled by conventional therapy.

Pegvaliase

  • Pegylated recombinant bacterial Anabaena variabilis phenylalanine ammonia lyase
  • Subcutanous daily injection, 5-60 mg
  • 51.1% decrease in blood Phe levels after 12 months of therapy
  • Half of patients on treatment achieve blood Phe level < 120 µmol/L, the upper limit of normal range, within 2 years
  • Adverse events (common): arthralgia (70.5%), injection-site reaction (62.1%), headache (47.1%)
  • Adverse events (severe): anaphylaxis (4.6%)
  • Current treatment: dietary phenylalanine restriction, sapropterin in THB-responsive PKU

Continue reading “Novel Enzyme Therapy Approved for Genetic Disease Phenylketonuria | FDA”

Platelet-Boosting Drug Approved to Prevent Surgical Bleeding in Cirrhosis Patients | FDA

The US FDA has approved the thrombopoietin receptor agonist avatrombopag (Doptelet®, AkaRx Inc) for the prevention of bleeding events in patients with chronic liver disease and thrombocytopenia undergoing a planned medical or dental procedure.

Avatrombopag

  • Thrombopoietin receptor agonist, stimulates platelet production
  • Decreases serious bleed risk 2-3 fold in patients w/ severe thrombocytopenia (<50*109/L) and chronic liver disease undergoing invasive procedures for up to 7 days after procedure
  • Most common adverse events: fever, abdominal pain, fatigue, edema, nausea
  • Current treatment: pre-procedural platelet transfusion 
  • May increase risk of thromboembolism in some patients

Continue reading “Platelet-Boosting Drug Approved to Prevent Surgical Bleeding in Cirrhosis Patients | FDA”

Chelating Agent ZS-9 Approved for Hyperkalemia | Medscape

The US FDA has approved the cation chelator sodium zirconium cyclosilicate (ZS-9, Lokelma®) for the treatment of hyperkalemia.

Sodium Zirconium Cyclosilicate

  • Oral potassium cation-exchange agent, not systemically absorbed
  • Onset of action 1 hour, median 2.2 hours until normokalemic
  • Not approved for life-threatening potassium elevations at this time
  • Daily dosing for 1 year twice as likely as placebo to maintain normokalemia
  • Most common adverse effects: GI distress, hypokalemia, UTI, edema
  • Adverse effects less frequent than patiromer (alternative chelating agent for hyperkalemia)

Continue reading “Chelating Agent ZS-9 Approved for Hyperkalemia | Medscape”