New Drugs and Devices 2009
Arzerra for Chronic Lymphocytic Leukemia
Arzerra (Ofatumumab) — GSK — this newly approved drug has been approved for the treatment of fludarabine-resistant chronic lymphocytic leukemia (CLL). In a study involving 138 patients, a partial response rate of 58% was observed in those with double resistant (fludarabine and alemtuzumab) CLL and 47% in those who had lymphatic bulky tumor masses. The progression-free survival was 5.7 mo. and 5.9 months respectfully. The most common adverse effects were infusion related reactions such as fever, diarrhea,rash,fatigue. Less common were upper respirators tract infections, pneumonia, anemia, and difficulty breathing.
ATryn for hereditary antithrombin deficiency.
ATryn (Anti-thrombin recombinant) — GTC Biotherapeutics — ATryn has been approved for the prevention of blood clots in patients with hereditary antithrombin deficiency (HAD). This new drug provides additional antithrombin to those patients with low antithrombin blood levels. It is of particular value in patients with HAD who are undergoing surgery, childbirth or prolonged bedrest. Only 1/31 patients with a HAD and a history of blood clots in at risk situations developed clots. Side effects were seen in 5% of injections and consisted of hemorrhage and reactions at the injection site.
Benzyl alcohol for head lice
Benzyl alcohol lotion — Sciele — this new topical preparation has been approved for the treatment of head lice infestations in children 6 months and older. Benzyl alcohol acts by smotherig them in 10 minutes of exposure to vbenzyl alcohol lotion. Two weeks after treatment >75% of patients remained lice-free.
Berinert for Hereditary Angioedema
Berinert (C-1 Esterase Inhibitor) — CSL — actions and pharmacology similar to Cinryze.
Cervarix for prevention of cervical cancer
Cervarix (Human papillomavirus bivalent (Types 16 and 18) — Glaxo — This new vaccine joins Gardasil (MSD) as an effective prevention of the development of cervical pre-cancers. Clinical studies have demonstrated this new vaccine to be 93% effective in preventing the development of cervical adenocarcinoma in situ. Virus Types 16 and 18 repesent 75% of the cervical cancers occurrung in the United States. The vaccination is to be used in women between 10 and 25 years old and is given in a 3 dose schedule to be completed within 6 months of the first dose.
Cinryze for Hereditary Angioedema
Cinryze (C1-Esterase inhibitor) — Viropharma — this has been approved for the prophylaxis of attacks of hereditary angioneurotic edema (HAE). HAE is a potentially lethal condition mimicking a hypersensitivity reaction. It may,at its severest, be potentially life-threatning by producing edema of the throat and difficult breating. Cinryze is given prophylactially every 3-4 days to patients with a history of HAE. The most common adverse effects were: upper respiratory tract infections, sinusitis, rash and headache.
Coartem for P. Falciparum malaria
Coartem (Artemether-Lumefantrine) — Novartis — this is a significant contribution to the therapy approved for use in treating p. Falciparum malarial infections. It is available as tablets taken by mouth It may be used both in children (>5kg) and adults. A standard 3 day treatment results in a cure rate of 95%.
Cycloset for diabetes
Cycloset (Bromocriptine) — Veroscience — this new drug taken by mouth has been approved for use in the treatment of type 2 diabetes. Although its mechanism of action is not known, it may act by increasing dopamine activity thus "reseting" the biological clock to improve metabolism problems related to diabetes. In one clinical trial 39% of patients taking Cycloset met the HbA1c goal compared with 11% of those taking a placebo. Gastrointestinal side effects resulted in 24% of patients discontinuing the drug.
Effient for acute coronary syndrome
Effient (Prasugrel) — Lilly — this new drug combined with percutaneous coronary intervention (PCI) is indicated for the acute treatment of coronary syndromes (acute coronary artery insufficiency). When taken with aspirin the risk of cardiovascular death, non-fatal heart attacks and non-fatal stroke decreased by 19% more than the current standard of therapy, clopidogrel(Plavix) although the incidence of bleeding, the most significant side effect, was increased. The overall risk of cardiovascular death and increased stroke was similar between the two groups.
Fanapt for schizophrenia
Fanapt (Iloperidone) — Pantheon — Fanapt has been approved for the acute treatment of schizophrenia in adults. Given by mouth, its mechanism of action is thought to be related to the fact it is a n=mixed dopamine D2/serotoinin 5ht2a antagonist. In a large trial Fanapt was significantly better than placebo in ameliorating schizophrenic symptoms. Adverse effects were relatively mild and and consisted of dizziness, dry mouth, nasal congestion, drowsiness, and weight increase.
Feraheme for chronic kidney disease iron deficiency anemia
Feraheme (Ferumoxytol) — Amag Pharms — this new injectable product has been approved specifically for the treatment of iron deficiency anemia associated with chronic kidney disease. Each 30 ml vial contains 510 mg elemental iron complex. It is administered intravenously and repeated 3-8 days later. In clinical trials 70% of patients had an increase in hemoglobin, TSAT and ferritin. The most common side effects were nausea (3.1%), dizziness (2.6%) and low blood pressure (2.5%).
Folotyn for refractory peripheral t-cell lymphoma
Folotyn (Pralatrexate) — Allos Therapeutics — This newly approved injectable drug is the first approved for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL). Patients with PTCL develop refractoriness to therapy very quickly. In a trial involving 115 patients wit PTCL Folotyn produced a 27% response rate. with a median duration of response of 9.4 months. Significant side effects were: thrombocytopenia (low platelets), sores in mouth (mucositis), neutropenia, and anemia.
Gelnique for overactive bladder
Gelnique (Oxybutin) — Watson — this is a new route of adminstration for this medication which in oral form is used to treat overactive bladder (OAB). Although significantly more expensive than the oral tablets because it does not require liver metabolism and is absorbed directly into the blood stream it results in fewer side effects such as dry mouth and constipation.
Intuniv for ADHD
Intuniv (Guanfacine) — Shire — this is an extended-release form of the guanfacine, a old drug used to treat hypertension. Despite its wide off-label use in ADHD this is the first time it has been officially approved for ADHD. Intuniv is given once daily and in 2 large studies produced a significant improvement in symptoms of ADHD beginning 1-2 weeks after therapy was instituted.
Istodax for Cutaneous T-Cell Lymphoma
Istodax (Romidepsin) — Gloucester Pharmaceuticals — This drug recently approved for the systemc treatment of cutaneous T-cell lymphoma (CTCL). Istodax is given by intravenous infusion every 7 days for 3 weeks each monthly cycle. Clinical studies demonstrated a 34-35% objective response rate (partial and complete) in patients who had become resistant to other systemic and cutaneous treatments. The most common adverse reactions in were nausea, fatigue, infections, vomiting and anorexia.
Multaq for atrial flutter and fibrillation
Multaq (Dronedarone) — Sanofi-Aventis — the approval of this drug represents a significant addition to those medications currently used to treat atrial fibrillation and flutter. It is specifically indicated for those patients with a recent episode of AF/AFL and associated cardiovascular risk factors (i.e., age >70, hypertension, diabetes, prior cerebrovascular accident, left atrial diameter ≥50 mm or left ventricular ejection fraction [LVEF] <40%), who are in sinus rhythm or who will be cardioverted. In clinical trials, premature discontinuation because of adverse reactions occurred in 11.8% of the dronedarone-treated patients and in 7.7% of the placebo-treated group. The most common reasons for discontinuation of therapy with MULTAQ were gastrointestinal disorders (3.2 % versus 1.8% in the placebo group) and QT prolongation (1.5% versus 0.5% in the placebo group).
Pennsaid for osteoarthritis of the knee
Pennsaid (Diclofenac) — Nuvo Research — This is a topical preparation of diclofenac, a non-steroidal anti-inflammatory drug. It is specifically indicated for osteoarthritis of the knee.
Raptiva withdrawn from market
Raptiva (Efalizumab)-has been withdrawn from the United states market because of the risk of progressive multifocal encephalopathy.
RiaSTAP for congenital fibrinogen deficiency
RiaSTAP (Fibrinogen Concentrate (Human) — CSL Behering — This newly approved drug is indicated for the treatment of congenital fibrinogen deficiency (CFD). Patients with CFD have a difficult time forming clots and are at risk for hemorrhage particularly after any traumatic procdure. In a study of 15 patients, once the target level of fibrinogen was achieved in the blood, 14/15 showed increased maximum clot firmness, a marker used to predict potential clinical benefit.
Sabril for infantile spasms
Sabril (Vigabatrin) — Lundbeck — Sabril has been approved for the treatment of spasms in infants ages one month-two years old. Approximately 16% of infants treated with Sabril became seizure free. Sabril is also indicated as adjunctive therapy for adult patients with refractory complex partial seizures (CPS) who have inadequately responded to several alternative treatments. Because of a sdignificant potential for retinal toxicity Sabril distribution in the United States is highly restricted.
Saphris for schizophrenia
Saphris (Asenapine) — Schering-Plough — this new chemical entity is indicated for the treatment of schizophrenia and bipolar I disorders in adults. Saphris in not indicated for the treatment of behavioral problems in older adults. The most common side effects include:inability to sit still or remain motionless, decreased oral sensitivity, and drowsiness.
Savella for fibromyalgia
Savella (Milnicipran) — Forest Labs — Savella has been approved for the treatment of fibromyalgia. It belongs to a class of drugs called serotonin and norepinephrine uptake inhibitors, approved for use as antidepressants. In studies comparing Savella with a placebo, a greater proportion of patients reported at least a 30% reduction and described themselves at much to very much improved. Side effects were: constipation, hot flush, sweating, vomiting, increased heart rate, dry mouth, and increases in blood pressure.
Simponi for rheumatoid arthritis
Simponi (Golimumab) — Centocor — this newly approved drug for rheumatoid arthritis is given once monthly,a significant advantage over other similar drugs on the market. It is also, as the other drugs in its class, acts by blocking an tumor necrosis factor (Anti-TNF). In a Phase 3 trial, Simponi significantly improvied the symptoms of ankylosing spondylitis and arthritic symptoms in patient with rheumatoid arthritis. The most dangerous side effect is suppression of the immune system which predisposes patients receiving Simponi to viral, bacterial, and fungal infection.
Stelara for Psoriasis
Stelara (Ustekinumab) — Janssen-Cilag — this newly approved drug is a significant addition to drugs currently used to treat moderate-severe psoriasis. It is indicated for those patients resistant to or poorly responsive to current standard therapies. A significant advantage is that it is given by injection every 12 weeks. Comparative studies have shown that after 12 weeks, Stelara was more effective than Enbrel which until now, was the therapy standard for moderate and severe psoriasis. In placebo-controlled trials by week 12, Stelara produced significant, visible improvement. The most common side effects included: joint pains, cough, headache, and upper respiratory tract infections.
Uloric for Hyperuricemia
Uloric (Febuxostat) — Takeda — this newly approved tablet has been approved for the treatment of hyperuricemia associated with gout. It acts by inhibiting the enzyme xanthine oxidase, a pharmacology similar to the only other drug used to treat hyperuricemia, allopurinol. Clinical studies have shown that Uloric at the 80 mg dose is 2X more effective than allopurinol in reducing serum uric acid level with 67% of patients achieving an end point of <6.0 mg/dl. Side effects are generally mild and include:liver function trst abnormalities,nausea,joint pain and rash.
Vectical for Psoriasis
Vectical (Calcitriol) — Galderma — this new topical ointment has been approved for mild to moderate psoriasis. I can be used on sensitive skin areas. Premarketing results were modest with clearing or regression to minimal lesions were observed in 34% of patients compared to 22% on the ointment vehicle only.
Vibativ for complicated skin infections
Vibativ (Telavancin) — Theravance/Astellas — this newly approved antibiotic is indicated for the treatment of adult patients with complicated skin infections caused by gram positive bacteria including methicillin-resistant staphylococcal infections. Vibativ is given I.V. once daily. The most common adverse effects are: taste disturbance, nausea, vomiting and foamy urine. More serious events seen in 5% of treated patients were cardiac, respiratory and infectious events.
Votrient for Advance Kidney Cancer
Votrient (Pazopanib) — Glaxo — Votrient joins others in the class of antineoplastics that act by preventing angiogenesis thus starving the tumor of the blood supply necessary for its growth. In a large study of 435 patients with advanced renal cell carcinoma the time before tumor regrowth or death was 9.2 months in the patients who took Votrient compared with 4.2 months for those who did not. Side effects reported were nausea,increase in blood pressure, abdominal pain and vomiting, and headache.
Zirgan for acute herpetic keratitis
Zirgan (Ganciclovir) — Sirion Therapeutics — Keratitis due to herpes simplex virus is a significant cause of corneal blindness. Characterized by reccurences, repeated exacerbations lead to corneal scarring and significant impairment of vision. This gel preparation resulted in clinical resolution in 7 days in 77% of patients. This compares favorably with acyclovir which resulted in a 72% resolution rate.