Specialty drugs Alexion Pharmaceuticals

1 specialty drugs

1.1 soliris
1.2 strensiq
1.3 kanuma

specialty drugs

alexion has employed strategy of developing drugs combat rare diseases. since targeted user base small such drugs, clinical drugs tend quicker , cheaper mass market drugs. additionally, big pharmaceutical companies have tended ignore these markets, creating niche minimal competition alexion. insurance companies have been willing pay high prices such drugs; since few of customers need drugs, high price not impact insurance companies outlays. success of specialty drugs rare diseases comes @ time when traditional drug business of selling medicines masses in decline...specialty drugs have gotten more expensive imagined.

soliris

alexion s first drug, soliris, first launched in 2007, used treat rare disorders atypical hemolytic uremic syndrome (ahus) , paroxysmal nocturnal hemoglobinuria (pnh). has been approved use in canada, european union, japan, , united states; however, availability in canada limited. in canada, access drug through private clinics; groups such canadian association of pnh patients lobbying change that. drug costs $450,000 year, , considered world s expensive drug. price of drug high few individuals can pay price. result, alexion hires public relations firms families institute campaigns pressure governments pay drug. alexion putting pressure on governments receive payments public purse. prices charged have high margin above cost price. in addition, of research development of soliris originates publicly funded universities. there ethical question pricing of drug , ethics of drug manufacturer. alexion on way developing second high price , high margined drug.

in september 2011, fda officially approved use of soliris treatment atypical hemolytic-uremic syndrome in both adults , children. more half of people ahus end dying of result of damage vital organs/organ failure (usually involving kidneys) caused uncontrolled complement activation. fda s decision grant approval received positive response medical community director of pediatric nephrology @ atlanta s children s hospital calling important advance has been made patients , families disease .

by 2010 soliris considered expensive drug in world. costs £340,200 per year treatment in uk , $500,000 year in canada. , us$409,500 year in united states (2010). in case of rarest diseases afflict fewer 10,000 people, biotech companies own approved drugs treat diseases can charge pretty whatever want. before testing soliris pnh, alexion tested drug rheumatoid arthritis, afflicts 1 million americans. trials failed. if had worked arthritis, alexion have had charge lower price use, have compete against drugs cost mere $20,000. alexion started selling soliris in 2008 making $295 million in 2007 stock price rising 130% in 2010.

in april , may 2013, controversy arose in belgium when media revealed government had refused pay seven-year-old boy s treatment because soliris expensive. boy s medicine cost 9,000 euros every 2 weeks. on may 4, 2013, de standaard reported press relations (pr) agency working alexion had helped boy s parents communicate story press. reported parents had believed benefactor dutch organization patients, , pr agency acted permission alexion. several politicians stated company attempting blackmail government, charges alexion denied. may 7, 2013 agreement had been reached reimburse medicine.

pharma, belgian pharmaceutical industry s association, opened internal investigation affair, possible breach of association s ethical standards alexion. however, on june 12, alexion received court gag order against pharma, preventing communicating investigation. @ same time, pharma opened court case against alexion pharma belgium. gag order revoked end of september 2013, case still pending in march 2015.

in october 2017 fda approved use of soliris treat adult patients generalized myasthenia gravis (gmg). in november 2017 company received patent soliris japanese patent office.

strensiq

in october 2015, alexion s second drug, strensiq (asfotase alfa), approved fda. used treat hypophosphatasia, rare metabolic disorder.

kanuma

kanuma, alexion acquired in acquisition of synageva, approved in 2015 treat lysosomal acid lipase deficiency, fatal genetic disorder cause fatty material build in blood vessel walls, liver, , other tissues. alexion estimates drug have annual sales of more $1 billion.