New Drug Approvals 2014

United States drug approvals and selected news stories. Data sourced to FiercePharma.com, FierceBiotech.com, company annual reports, FDA unless otherwise noted. See: http://www.letsthinkabout.us/post/2014-fda-drug-approvals for other information

Transcript

2. A record year for approvals, second highest since 53 in 1996 1
3. 41 new drugs (up from 29 in 2013) 17 New first-in-class therapies Merck’s Belsomra® (suvorexant)- first orexin receptor antagonist, control schedule IV for insomnia Merck’s Keytruda® (pembrolizumab)- breakthrough chemotherapy first indicated for non-resectable melanoma. Currently undergoing trials for lung and renal cancers. (blockbuster) Roche’s Esbriet® (prifenidone)- first treatment for idiopathic pulmonary fibrosis Gilead’s Harvoni® (ledipasvir/sofosbuvir)- first combination, once-daily treatment for genotype-1 hepatits C virus Knight’s Impavido® (miltefosine)- first treatment for rare parasitic disease visceral and cutaneous leshmaniasis Anacor’s Kerydin® (tavaborole)- first treatment for onchomycosis of the toenails, fungal infection AstraZeneca’s Lynparza® (olaparib)- likely a second-line therapy for patients who have failed other therapies AstraZeneca’s Myalept® (metreleptin)- Orphan drug for treating patients with partial lipodystrophy due to metabolic disorders Biogen Idec’s Plegridy®- (peginterferon beta-1a)- long-acting version of Avonex dosed every 2 weeks vs once weekly Chelsea Therapeutic’s Northera® (droxidopa)- for rare neurogenic orthostatic hypotension. Has block-box warning for stroke as it elevated blood pressure while patient lies down Boehringer Ingelheim’s Ofev® (esbriet)- first treatment for idiopathic pulmonary fibrosis Celgene’s Otezla® (apremalist)- first oral therapy for psoriatic arthritis and psoriasis Johnson & Johnson’s Sylvant® (siltuximab)- orphan drug for multicentric Castleman’s disease, characterized by abnormal growth ofimmune cells in lymph nodes AbbVie’s Viekira Pak® (ombitasvir/paritaprevir/dasabuvir/ritonavir)- multi-daily-dose regimen hepatitis C drug that is first-in-class for mechanism of action Biomarin’s Vimizim® (elosulfase alfa)- for the rare enzyme deficiency Morquio A. Affects only 3000 patients in the developed world, 800 in US Merck’s Zontivity® (vorapaxar)- anticoagulant of PAR-1 antagonist. Has black box warning for excessive bleeding Genzyme’s Lemtrada® (alemtuzumab) a CD52 directed cytolytic monoclonal antibody indicated for the treatment of replasing forms of multiple sclerosis 2014 Pharmaceutical Marketplace Highlights 2
4. 41 new drugs (up from 29 in 2013) New Diabetes Therapies AstraZeneca’s Farxiga® (dapagliflozin)- SGLT-2 inhibitor. Has possible bladdar cancer ADE. Projected blockbuster Eli Lilly’s Trulicity® (dulaglutide)- GLP-1 agonist once weekly injection proved non-inferior to Novo Nordisk’s Victoza® for lowering A1c. Was not non-inferior in body-weight reduction GSK’s Tanzeum® (albiglutide)- GLP-1 agonist once weekly that did not prove non-inferiority to Victoza® in lowering A1c. Eli Lilly and Boehringer Ingelheim’s Jardiance® (empagliflozin)- SGLT-2 inhibitor that does not have the bladder cancer warning of Farxiga® nor the liver damage warning of Invokana® New Cancer Therapies Amgen’s Blincyto® (blinatumomab)- first bispecific T-cell engager for Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia Merck’s Keytruda® (pembrolizumab)- breakthrough chemotherapy first indicated for non-resectable melanoma. Currently undergoing trials for lung and renal cancers. (blockbuster) Eli Lilly’s Cyramza® (ramucirumab)- for advanced stomach cancer (blockbuster) Novartis’ Zykadia® (certinib)- for second line therapy of ALK- positive non-small cell lung cancer following treatment with Xlakori® New Cancer Therapies(cont.) Spectrum’s Beleodaq® (belinostat)- for peripheral T-cell lymphoma Gilead’s Zydelig® (idelasib)- for B-cell blood cancers (blockbuster) BMS’ Opdivo® (nivolumab)- Indicated for melanoma currently with lung cancer in pipeline. Same MoA as Keytruda® (blockbuster) AstraZeneca’s Lynparza® (olaparib) for ovarian cancer (blockbuster) New Antibiotics The Medicines Company’s Orbactiv® (oritavancin)- for acute skin infections caused by S aureus, Streptococcus and Enterococcus Cubist’s Zerbaxa® (ceftazolane and tazobactam)- For gram- negative infections (projected blockbuster) Cubist’s Sivextro® (tedizolid)- For skin infections in adults Actavis’ Dalvance® (dalbavancin)- for acute bacterial skin and skin structure infections Alcon’s Xtoro® (finafloxacin)- for acute otitis externa, an infection of the outer ear and ear canal known as swimmer’s ear New Influenza Antiviral BioCryst’s Rapivab® (peramivir)- for treatment of acute influenza. IV therapy with low projected sales 2014 Pharmaceutical Marketplace Highlights 3
5. New Antifungal Therapy Anacor’s Kerydin® (tavaborole)- for the treatment of onchomycosis in toenails, a difficult-to-treat, recurring fungal infection Valeant’s Jublia® (efinaconazole 10%) for onychomycosis of the toenails New Multiple Sclerosis Therapy Genzyme’s Lemtrada® (alemtuzumab) a CD52 directed cytolytic monoclonal antibody indicated for the treatment of replasing forms of multiple sclerosis Biogen Idec’s Plegridy®- (peginterferon beta-1a)- long- acting version of Avonex dosed every 2 weeks vs once weekly New Opioid-Induced Constipation Therapy AstraZeneca and Nektar’s Movantik® (naloxegol)- for patients who have opioid-induced constipation in adults with chronic noncancer pain. (blockbuster) 41 new drugs (up from 29 in 2013) New Hepatitis C Therapy Gilead’s Harvoni® (ledipasvir/sofosbuvir)- first combination, once-daily treatment for genotype-1 hepatits C virus. Estimated peak sales = $10 billion+ AbbVie’s Viekira Pak® (ombitasvir/paritaprevir/dasabuvir/ritonavir)- multi- daily-dose regimen hepatitis C drug that is first-in-class for mechanism of action New Imaging Agents Piramal Imaging’s Neuraceq®(florabetaben F18) for PET imaging fo rhte brain to estimate B-amyloid plaquedensity in adult patients with cognitive impairment who are being evaluated for Alzheimer’s Disease and other causes of cognitive decline. Lymphoseek® (technetium 99m tilmanocept) to help determine the extent of squamous cell carcinoma in head and neck region Bracco’s Lumason® (sulfur hexafluoride lipid microsphere)- used to visualize the left ventricle and inside of heart chambers more clearly. Originally submitted to FDA in 2001 2014 Pharmaceutical Marketplace Highlights 4
6. Invega® paliperidone- for Szchizoaffective disorder Avastin® (bevacizumab)- with chemotherpay for platinum resistant recurrent ovarian cancer and metastatic cervical cancer Imbruvica® (ibrutinib)- for Mantle Cell lymphoma and chronic lymphocytic leukemia Xolair® (omalizumab)- for Chronic Idiopathic Urticaria Topamax® (topiramate)- for migraine prevention in adolescents Nexium® (esomeprazole)- approved for over-the- counter 24 hours use Pradaxa® (dabigatran)- for DVT and pulmonary hypertension Zorvolex (diclofenac)- for manangement of osteoarthritis pain) Promacta® (eltrombopag)- for severe aplastic anemia Otezla® (apremilast)- for moderate to severe plaque psoriasis Ozurdex® (dexamethasone)- for treatment of diabetic macular edema Cyramza® (ramucirumab) for aggressive treatment of non0small cell lung cancerKalydeco® (ivacaftor) for people with cystic fibrosis ages 6 and older who have the R117H mutation Eliquis® (apixaban)- for treatment of deep vein thrombosis and pulmonary embolism Somatuline Depot® (lanreotide acetate)- for treatment of acromegaly and gastroenteropancreatic neuroendocrine tumors Existing Drugs with New Indications 5
7. Abilify Maintena® (aripiprazole)- dual- chamber syringe for schizophrenia Afreeza® (human insulin rDNA norigin) inhalation powder insulin Bellafill® (dermal filler) for facial wrinkles and acne scars Bunavail® (buprenorphine and naloxone) buccal film for maintenance treatment of opioid dependence Bydureon® (exenatide) once weekly injection Fluzone® (influenza virus vaccine, inactivated) an intradermal quadrivalent forumulation Hysingla ER® (hydrocodone) Extended release tablets with abuse deterrent Kalbitor® (ecallantide) –for adolescents with hereditary angiodedema Kitabis Pak® (tobramycin) inhalation solution co-packaged with PARI LC PLUS® reusable nebulizer for the management of cystic fibrosis Uceris® (budesonide) rectal foam for ulcerative colitis Xartemis® (acetaminophen and oxycodone) extended release tablets Zubsolv® (buprenorphine and naloxone)- approved with double dose Significant New Dosage Forms 6
8. Genzyme and Sanofi’s Cerdelega® (eligllustat) an oral treatment for Gaucher’s disease, which is a deficiency of the enzyme glucocerebrosidase and leads to fatty buildups in spleen and liver Roche’s Esbriet® (prifenidone)- first treatment for idiopathic pulmonary fibrosis Johnson & Johnson’s Sylvant® (siltuximab)- orphan drug for multicentric Castleman’s disease, characterized by abnormal growth of immune cells in lymph nodes Biomarin’s Vimizim® (elosulfase alfa)- for the rare enzyme deficiency Morquio A. Affects only 3000 patients in the developed world, 800 in US Chelsea Therapeutic’s Northera®(droxidopa)- for rare neurogenic orthostatic hypotension. Has block-box warning for stroke as it elevated blood pressure while patient lies down AstraZeneca’s Myalept® (metreleptin)- Orphan drug for treating patients with partial lipodystrophy due to metabolic disorders Knight’s Impavido® (miltefosine)- first treatment for rare parasitic disease visceral and cutaneous leshmaniasis Boehringer Ingelheim’s Ofev® (esbriet)- first treatment for idiopathic pulmonary fibrosis 7
14. 13 Select Other News
15. bernard.hsu@allgoodpeople.us 14