AstraZeneca Leading in New Drug Approvals in 2014


From DCAT Value Chain Insights (VCI)

By Patricia Van Arnum posted 05-13-2014 15:19

  

As the pharmaceutical industry approaches the halfway mark in 2014, how is the industry faring for approvals of new molecular entities and biologics license applications? Which companies have earned coveted FDA approval? DCAT Value Chain Insights takes a look at the key approvals thus far in 2014.

As the pharmaceutical industry nears the halfway mark, 2014 is shaping up to be on par with 2013 in terms of the number of new drug approvals as measured by approval by FDA’s Center for Drug Evaluation and Research of new molecular entities (NMEs) and therapeutic biologics filed as original biologics license applications (BLAs). Thus far in 2013, 13 NMEs and BLAs have been approved by CDER (see Table I). In all of 2013, 27 NMEs and BLAs were approved. But the numbers alone do not tell the whole story. Reflecting the move to more specialized drug development, seven of the 13 new drugs approved are orphan drugs (defined by FDA as drugs that treat diseases that affect 200,000 or fewer Americans) with one drug a breakthrough therapy, a new designation by FDA to expedite drug review for drugs that offer substantial improvement over existing drugs. Among the large pharmaceutical companies, AstraZeneca led the way with two approvals, and Eli Lilly, GlaxoSmithKline (GSK), Johnson & Johnson’s Janssen Biotech, Merck & Co., and Novartis each scored at least one new approval.

 

Winners thus far in 2014
Of the large pharmaceutical companies, AstraZeneca led the pack with two FDA approvals in 2014: the diabetes drug Farxiga (dapaglifozin) and the orphan drug Myalept (metreleptin), a recombinant analog (laboratory-created form) of human leptin as a replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy.

Farxiga was approved by FDA in January 2014. It was approved in Europe, where it is marketed as Forxiga, in November 2012 and in Japan in March 2014. Farxiga is the second drug approved by FDA in a new class of drugs to treat diabetes, sodium glucose cotransporter 2 (SGLT2) inhibitors.The first was Janssen Pharmaceuticals’ Invokana (canagliflozin), which was approved in 2013. As a SGLT2 inhibitor, Farxiga blocks the reabsorption of glucose by the kidney, increases glucose excretion, and lowers blood glucose levels. It indicated as an adjunct to diet and exercise as a once daily oral medication to improve glycemic control in adult patients with Type 2 diabetes mellitus as an add-on combination therapy or as monotherapy in metformin-intolerant patients.

AstraZeneca gained full rights to Farxiga as well as Myalept as part of its $2.7-billion acquisition of its diabetes alliance with Bristol-Myers Squibb, a deal which was completed in February 2014. The acquisition gave AstraZeneca ownership of the intellectual property and global rights for the development, manufacture, and commercialization of the diabetes business, which included Farxiga/Forxiga, Xigduo (dapaglifozin and metformin HCl extended release), Onglyza (saxagliptin), Komboglyze (saxagliptin and metformin HCl), Kombiglyze XR (saxagliptin and Byetta (exenatide), Bydureon (exenatide extended-release for injectable suspension), metreleptin, and Symlin (pramlintide acetate). In addition to the $2.7-billion, AstraZeneca has also agreed to pay up to $1.4 billion in regulatory, launch, and sales payments, and various sales-related royalty payments up until 2025, $600 million of which relates to the approval of Farxiga in the US. In addition, AstraZeneca may make payments of up to $225 million when certain assets are subsequently transferred. AstraZeneca is working to complete the transfer of the BLA for Mylaept from Bristol-Myers Squibb Company to AstraZeneca as part of the acquisition of the diabetes alliance assets, including Myalept and Amylin Pharmaceuticals, which was completed on February 1, 2014. Bristol-Myers Squibb had acquired Amylin Pharmaceuticals in 2012.

Other pharmaceutical companies have late-stage SGLT2 inhibitors in development. Pfizer and Merck & Co are developing their SGLT2 inhibitor, ertugliflozin, which is in Phase III trials. In April 2013, Pfizer and Merck formed a collaboration agreement for the development and commercialization of Pfizer’s ertugliflozin. The companies are collaborating on the clinical development and commercialization of ertugliflozin and ertugliflozin-containing fixed-dose combinations with metformin and Merck’s Januvia (sitagliptin) tablets. Merck will continue to retain the rights to its existing portfolio of sitagliptin-containing products. Pfizer received an upfront payment and milestones of $60 million and will be eligible for additional payments associated with the achievement of pre-specified future clinical, regulatory, and commercial milestones. Merck and Pfizer will share potential revenues and certain costs on a 60/40 percent basis.

Boehringer Ingelheim and Eli Lilly are partnered for the development of their SGLT2 inhibitor, empagliflozin. In March 2014, FDA issued a Complete Response Letter for the companies’ new drug application (NDA) for empagliflozin. The Complete Response Letter referenced previously observed deficiencies at a Boehringer Ingelheim facility where empagliflozin will be manufactured. FDA stated these deficiencies need to be resolved before the approval of the application. FDA has not asked Boehringer Ingelheim to complete any new clinical trials to support the approval of the application. In its first-quarter earnings release, Eli Lilly said it and Boehringer Ingelheim expect FDA action in 2014. In March 2014, the companies received a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA), recommending approval of empagliflozin, as an adjunct to diet and exercise to improve glycemic control, or blood glucose levels, in adults with Type 2 diabetes. And in April 2014, the companies reported that FDA had accepted their NDA for a combination tablet of empagliflozin and linagliptin for the treatment of adults with Type 2 diabetes. If granted approval by FDA, the combination would offer the mechanisms of action of a SGLT2 inhibitor and a dipeptidyl peptidase-4 (DPP-4) inhibitor. SGLT2 inhibitors remove excess glucose through the urine by blocking glucose re-absorption in the kidney. DPP-4 inhibitors work by increasing hormones that stimulate the pancreas to produce more insulin and stimulate the liver to produce less glucose.

In January 2014, Astellas Pharma received marketing approval in Japan for its SGLT2 inhibitor Suglat (ipragliflozin L-proline). In April 2014, the Japanese pharmaceutical company Taisho Pharmaceutical received manufacturing and marketing approval in Japan for its SGLT2 inhibitor Lusefi (luseogliflozin) for treating Type II diabetes.

Among smaller companies, Islet Sciences, a biopharmaceutical company based in Raleigh, North Carolina, announced a letter of intent to acquire BHV Pharma, based in Research Triangle Park, North Carolina, which is developing the SGLT2 inhibitor remogliflozin etabonate for the treatment of Type II diabetes. Lexicon Pharmaceuticals, Inc., based in Princeton, New Jersey, has an SGLT2 inhibitor, LX4211,in Phase II clinical trials.

Other new drug approvals
Among the other large pharmaceutical companies, five companies—Eli Lilly, GSK, Johnson & Johnson’s Janssen Biotech, Merck & Co., and Novartis—each had either an NME or original BLA approved by FDA’s CDER thus far in 2014.

GSK received approval for its diabetes drug Tanzeum (albiglutide) in April 2014. Albiglutide, a glucagon-like peptide-1 (GLP-1) receptor agonist is a biological product for treating Type 2 diabetes, administered once-weekly using an injector pen supplied with a 5-mm 29-gauge thin-walled needle. It is a recombinant fusion protein comprised of two tandem copies of modified human GLP-1 genetically fused in tandem to human albumin. Glucagon-like peptide-1 is an important incretin hormone that helps reduce blood glucose levels but, in people with Type 2 diabetes, its production is often reduced or absent, according to a GSK press release. GSK anticipates the US launch of Tanzeum in the third quarter of 2014. Albiglutide was licensed by the EMA in March 2014, under the brand name Eperzan.

In gaining approval for Tanzeum, GSK will be competing against other GLP-1 agonists: AstraZeneca’s Byetta (exenatide injection) and Bydureon (exenatide extended-release injectable suspension) and Novo Nordisk’s Victoza (liraglutide). Sanofi’s Lyxumia (lixisenatide) was in-licensed from Zealand Pharma A/S and is approved in Europe for the treatment of adults with Type 2 diabetes mellitus to achieve glycemic control in combination with oral glucose-lowering medicinal products and/or basal insulin when these, together with diet and exercise, do not provide adequate glycemic control. Lyxumia is also approved in Mexico, Australia, Japan, and Brazil for the treatment of adults with Type 2 diabetes. Sanofi plans to resubmit the new drug application for lixisenatide in the United States in 2015, after completion of the ELIXA cardiovascular outcomes study.

Merck & Co gained approval last week for the anti-platelet agent Zontivity (vorapaxar). Zontivity is the first in a new class of drugs, called protease-activated receptor-1 (PAR-1) antagonists. It is designed to decrease the tendency of platelets to clump together to form a blood clot. By decreasing the formation of blood clots, Zontivity decreases the risk of heart attack and stroke. It was approved by FDA to reduce the risk of heart attack, stroke, cardiovascular death, and the need for procedures to restore the blood flow to the heart in patients with a previous heart attack or blockages in the arteries to the legs.

Merck also received approval for two immunotherapies by FDA’s Center for Biologics Evaluation and Research: Grastek (Timothy grass pollen allergen extract), an allergen extract for the treatment of grass pollen-induced allergic rhinitis and Ragwitek (short ragweed pollen allergen extract indicated as immunotherapy for short ragweed pollen-induced allergic rhinitis. CBER regulates and approves allergenics, blood and blood products, cellular and gene therapy products, tissue and tissue products, vaccines, and xenotransplantation. CDER approves NMEs and therapeutic biologics filed as BLAs.

Novartis received approved for its cancer drug Zykadia (ceritinib), designed to treat a certain type of metastatic non-small cell lung cancer (NSCLC). Zykadia is an anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor that blocks proteins that promote the development of cancerous cells. It is intended for patients with metastatic ALK-positive NSCLC who were previously treated with crizotinib, the only other approved ALK tyrosine kinase inhibitor. Zykadia is the fourth drug with breakthrough therapy designation to receive FDA approval and the first one to do so in 2014. The Food and Drug Administration Safety and Innovation Act, passed in July 2012, gave FDA the ability to designate a drug a breakthrough therapy at the request of the sponsor if preliminary clinical evidence indicates that the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. A breakthrough therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug-development program. 2013 was the first year any new drug was approved with the breakthrough designation when three drugs received breakthrough therapy designation (see Table II).

Eli Lilly and Janssen Biotech each received approval of an orphan drug. Eli Lilly received FDA approval for Cyramza (ramucirumab) to treat advanced stomach cancer or gastroesophageal junction adenocarcinoma. Janssen Biotech received FDA approval for Sylvant (siltuximab), a drug to treat patients with multicentric Castleman’s disease.

Chelsea Therapeutics received approval for its orphan drug Northera (droxidopa) for the treatment of neurogenic orthostatic hypotension (NOH). NOH is a rare, chronic drop in blood pressure upon standing that is associated with Parkinson's disease, multiple-system atrophy, and pure autonomic failure, dopamine beta hydroxylase deficiency, and non-diabetic autonomic neuropathy. Northera is expected to be launched in the second half of 2014. Earlier this month, H. Lundbeck A/S (Lundbeck)  agreed to acquire  Chelsea Therapeutics for $658 million.  

A look at 2013
The NMEs/BLA approvals by CDER in 2013 reflect the increased specialization of drug development with nine drugs approved as orphan drugs as well as the strategy to utilize different regulatory mechanisms, such as FDA’s new breakthrough therapy designation as well as priority review and accelerated approval, to expedite drug review (see Table II). Almost half (48%) of the 27 NMEs approved in 2013 (13 of 27) were designated in one or more categories of fast track, breakthrough therapy, priority review, and/or accelerated approval. These approaches also encourage greater innovation as one-third or nine of the 27 NMEs/BLAs approved by FDA’s CDER in 2013 were identified by FDA as first-in-class, meaning drugs which, for example, use a new and unique mechanism of action for treating a medical condition (1). First-in-class is one indicator of the innovative nature of a drug. Noteworthy first-in-class products approved in 2013 included: Janssen Pharmaceuticals’ Invokana (canagliflozin) for treating Type 2 diabetes; Roche/Genentech’s Kadcyla (ado-trastuzumab emtansine), an antibody drug conjugate to treat HER2-positive late-stage (metastatic) breast cancer; Gilead Sciences’ Sovaldi (sofosbuvir), an interferon-free oral treatment option for treating chronic hepatitis C virus (HCV) infection; and GSK’s Mekinist (trametinib dimethyl sulfoxide) for treating metastatic melanoma. Other first-in-class drugs that received approval in 2013 were: Bayer’s Adempas (riociguat); Pharmacyclics/Janssen Biotech’s Imbruvica (ibrutinib); Sanofi/Genzyme’s Kynamro (mipomersen sodium); Biogen Idec’s Tecfidera (dimethyl fumarate); and Bayer’s Xofigo (radium RA-223 dichloride) (see Table II) (1).

In 2013, one third or nine of the 27 NMEs/BLAs approved by FDA were orphan drugs. Among the nine approvals by CDER in 2013, seven drugs were from the large pharmaceutical companies: Bayer’s Adempas (riociguat), Boehringer Ingelheim’s Gilotrif (afatinib dimaleate), Genentech/Roche’s Gazyva (obinutuzumab), Genzyme/Sanofi’s Kynamro (mipomersen sodium), two drugs by GSK, Mekinist (trametinib dimethyl sulfoxide) and Tafinlar (dabrafenib mesylate), and Janseen Biotech's /Pharmacyclics’Imbruvica (ibrutimib) (see Table II) (1).

Ten of the 27 NMEs approved in 2013 (37%) were designated by CDER as fast rack, meaning drugs with the potential to address unmet medical needs. Fast track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to developers and by enabling developers to use a “rolling review” process such that CDER can review portions of an application ahead of the submission of the full application. The fast-tracked approvals in 2013 were: Bayer’s Xofigo; Boehringer Ingelheim’s Gilotrif; Celgene’s Pomalyst; Gilead Sciences’ Sovaldi; GSK’s Mekinist and Tafinlar; Janseen Biotech's /Pharmacyclics’ Imbruvica and Janssen Pharmaceuticals’ Olysio; Roche/Genentech’s Kadcyla; and Viiv Healthcare’s Tivicay (1).

Three of the 27 NMEs approved in 2013 (11%) were designated by CDER as breakthrough therapies, meaning drugs with preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A breakthrough therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug-development program. Breakthrough status is designed to help shorten the development time of a promising new therapy and is a new designation that went into effect after July 9, 2012. 2013 was the first year any new drug was approved with the breakthrough designation. In 2013, the three drugs approved as breakthrough therapies were: Gilead Sciences' Sovaldi (sofosbuvir) to treat chronic hepatitis C virus infection, Janssen Biotech/Pharmacyclics' Imbruvia (ibrutinib) to treat mantle cell lymphoma, and Genentech/Roche’s Gazyva (obinutuzumab) to treat chronic lymphocyte leukemia (1).

Ten of the 27 NMEs approved in 2013 (37%) were designated priority review, in which CDER determines the drug to potentially provide a significant advance in medical care and sets a target to review the drug within six months instead of the standard 10 months (see Table II). Two of the 27 NMEs approved in 2013 (7%) were approved under FDA’s Accelerated Approval program, which allows early approval of a drug for serious or life-threatening illness that offers a benefit over current treatments. This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that FDA considers reasonably likely to predict clinical benefit. After this approval, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug (see Table II) (1).

Reference
1. FDA, 2013 Novel New Drugs Summary (January 2014).


Table I: New molecular entities (NMEs) and original biologics license applications (BLAs) approved by FDA's Center for Drug Evaluation and Research, 2014 (as of May 10, 2014)*

Company Proprietary Name
(Active Ingredient)


Dosage Form/Route of Administration Indication Special Designation
AstraZeneca Farxiga (dapaglifozin);NME Tablet/oral To improve glycemic control, along with diet and exercise, in adults with type 2 diabetes


AstraZeneca
Myalept (metreleptin); BLA
Injectable/Injection
To treat metabolic disorders
secondary to lipodystrophy

Orphan Drug
BioMarin Pharmaceutical
Vimizim (elosulfase alfa); BLA
Injectable/Injection
To treat Mucopolysaccharidosis Type IVA (Morquio A syndrome)

Priority Review; Orphan Drug
Celgene
Otezla (apremilast); NME
Tablet/oral
To treat adults with active psoriatic arthritis

 
Chelsea Therapeutics
Northera (droxidupa); NME
Capsule/Oral
To treat neurogenic orthostatic hypotension
Accelerated Approval; Priority Review; Orphan Drug

Eli Lilly
Cyramza (ramucirumab); BLA
Injectable/Injection
To treat advanced stomach cancer or gastroesophageal junction adenocarcinoma

Priority Review; Orphan Drug
GlaxoSmithKline
Tanzeum (albiglutide): BLA
Injectable/Injection
To improve glycemic control, along with diet and exercise, in adults with type 2 diabetes

 
Janssen Biotech
Sylvant (siltuximab); BLA
Injectable/Injection
To treat patients with multicentric Castleman’s disease

Priority Review; Orphan Drug 

Merck & Co.
Zontivity (vorapaxar); NME
Tablet/oral
To reduce the risk of heart attack, stroke, cardiovascular death, and need for procedures to restore the blood flow to the heart in patients with a previous heart attack or blockages in the arteries to the legs


Novartis
Zykadia (ceritinib); NME
Capsule/oral
To treat patients with a certain type of metastatic non-small cell lung cancer
Zykadia is the fourth drug with breakthrough therapy designation to receive FDA approval and first one to do so in 2014; Priority Review: Accelerated Approval

Paladin Therapeutics
Impavido(miltefosine); NME
Capsule/oral
To treat the tropical disease leishmaniasis
Fast track; Priority Review; Orphan Drug

Piramal Imaging
Neuraceq (florbetaben F 18 injection); NME
Solution/Intravenous
For positron emission tomography imaging of the brain


Vanda Pharmaceuticals
Hetlioz (tasimelteon); NME
Capsule/Oral
To treat non-24-hour sleepwake disorder in blind individuals without light perception
Priority Review; Orphan Drug
FDA Designations
Accelerated Approval: FDA’s Accelerated Approval program allows early approval of a drug for serious or life-threatening illness that offers a benefit over current treatments. This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that FDA considers reasonably likely to predict clinical benefit. After this approval, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug.

Breakthrough Therapy: The Food and Drug Administration Safety and Innovation Act, passed in July 2012, gave the FDA the ability to designate a drug a breakthrough therapy at the request of the sponsor if preliminary clinical evidence indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. A breakthrough therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. 2013 was the first year any new drug was approved with the breakthrough designation.

Fast Track: Fast Track is given to drugs with the potential to address unmet medical needs. Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to developers and by enabling developers to use a “rolling review” process such that CDER can review portions of an application ahead of the submission of the full application.

Orphan Drugs: Orphan Drugs are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment.

Priority Review: Priority Review is provided when FDA’s CDER determines the drug to potentially provide a significant advance in medical care and sets a target to review the drug within six months instead of the standard 10 months.

AstraZeneca is working to complete the transfer of the biologics license application for Myalept from Bristol-Myers Squibb to AstraZeneca as part of AstraZeneca's acquisition of the diabetes alliance, including Myalept, and Amylin Pharmaceuticals, which was completed on February 1, 2014.

As part of the sale of its diabetes alliance (excluding China) to AstraZeneca, Bristol-Myers Squibb received from AstraZeneca a payment of approximately $2.7 billion at closing and a subsequent milestone payment of $600 million for the US approval of Farxiga (dapagliflozin). The company also received $100 million in the second quarter for the approval of dapagliflozin in Japan. Under terms of the agreement, Bristol-Myers Squibb will potentially receive additional regulatory- and sales-based milestone payments from AstraZeneca of up to $700 million, royalty payments based on net sales through 2025 and additional payments if and when certain assets are subsequently transferred.

H. Lundbeck A/S agreed to acquire for $658 million Chelsea Therapeutics International on May 8, 2014.

Source: FDA


Table II: New molecular entities (NMEs) and original biologics license applications (BLAs) approved by FDA's Center for Drug Evaluation and Research, 2013.

Company Proprietary Name
(Active Ingredient)


Dosage Form/Route of Administration Indication Special Designation
Actelion Pharmaceuticals Opsumit (macitentan); NME Tablet/oral To treat adults with pulmonary arterial hypertension

Orphan Drug
Bayer Healthcare
Adempas (riociguat); NME
Tablet/oral
To treat adults with two forms of pulmonary hypertension

Orphan Drug; Priority Review 
Bayer Healthcare
Xofigo (radium RA-223 dichloride); NME
Solution/intravenous
To treat men with symptomatic metastatic castration-resistant prostate cancer that has spread to bones but not to other organs

Fast Track; Priority Review 
Biogen Idec
Tecfidera (dimethyl fumarate); NME
Capsule, delayed release/oral
To treat adults with relapsing forms of multiple sclerosis


Boehringer Ingelheim
Gilotrif (afatinib dimaleate); NME
Tablet/oral
To treat metastatic non-small cell lung cancer whose tumors express specific types of epidermal growth factor receptor gene mutation, as detected by an FDA-approved test

Orphan Drug; Fast Track; Priority Review

Celgene
Pomalyst (pomalidomide); NME
Capsule/oral
To treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs

Orphan Drug; Fast Track; Accelerated Approval
GE Healthcare
Vizamyl (flutemetamol F-18); NME
Injectable/intravenous
A radioactive diagnostic drug for use with positron emission tomography (PET) imaging of the brain in adults being evaluated for Alzheimer's disease and dementia

 
Gilead Sciences
Sovaldi (sofosbuvir); NME
Tablet/oral
To treat chronic hepatitis C virus infection

Was the third drug with breakthrough therapy designation to receive FDA approval; Fast Track; Priority Review
GlaxoSmithKline
Anoro Ellipta (umeclidinium and vilanterol inhalation powder); NME
Powder/inhalation
For the once-daily, long-term maintenance treatment of airflow obstruction in patients with chronic obstructive pulmonary disease


GlaxoSmithKline
Tafinlar (dabrafenib mesylate); NME
Capsule/oral
To treat patients with melanoma whose tumors express the BRAF V600E gene mutation

Orphan Drug; Fast Track

GlaxoSmithKline
Mekinist (trametinib dimethyl sulfoxide); NME
Tablet/oral
To treat patients whose tumors express the BRAF V600E or V600K gene mutations

Orphan Drug; Fast Track;

GlaxoSmithKline
Breo Ellipta (fluticasone furoate; vilanterol trifenatate); NME
Powder/inhalation
For the long-term, once-daily, maintenance treatment of airflow obstruction in patients with chronic obstructive pulmonary disease, including chronic bronchitis and/or emphysema


Guebert
Dotarem (gadoterate meglumine); NME
Solution/intravenous
For use in magnetic resonance imaging of the brain, spine and associated tissues of patients ages 2 years and older

Priority Review:
Janssen Biotech and Pharmacyclics
Imbruvica (ibrutinib); NME
Capsule/oral
To treat patients with mantle cell lymphoma, a rare type of blood cancer
Was the second drug with breakthrough therapy designation to receive FDA approval; Orphan Drug; Fast Track; Priority Review; Accelerated Approval
Janssen Pharmaceuticals
Invokana (canagliflozin): NME
Tablet/oral
Used with diet and exercise, to improve glycemic control in adults with Type 2 diabetes


Janssen Pharmaceuticals
Olysio (simeprevir sodium); NME
Capsule/oral
To treat chronic hepatitis C virus infection

Fast Track; Priority Review
Navidea Biopharmaceuticals
Lymphoseek Kit (technetium Tc-99m tilmanocept); NME
Injectable/injection
A radioactive diagnostic imaging agent that helps doctors locate lymph nodes in patients with breast cancer or melanoma who are undergoing surgery to remove tumor-draining lymph nodes

 
Pfizer
Duavee (bazedoxifene acetate; estrogens, conjugated); new combination
Tablet/oral
To treat moderate-to-severe hot flashes associated with menopause and to prevent osteoporosis after menopause

 
Roche/Genentech
Gazyva (obinutuzumab); BLA
Injectable/injection
For use in combination with chlorambucil to treat patients with previously untreated chronic lymphocytic leukemia
Was the first drug with breakthrough therapy designation to receive FDA approval; Orphan Drug; Priority Review

Roche/Genentech
Kadcyla (ado-trastuzumab emtansine); BLA
Vial/single use
For patients with HER2-positive metastatic breast cancer

Fast Track; Priority Review 
Sanofi/Genzyme
Kynamro (mipomersen sodium); NME
Solution/subcutaneous
To treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia

Orphan Drug
Shionogi
Osphena (ospemifene); NME
Tablet/oral
To treat women experiencing moderate to severe dyspareunia

 
Sunovion Pharmaceuticals
Aptiom (eslicarbazepine acetate); NME
Tablet/oral
As an add-on medication to treat seizures associated with epilepsy

 
Takeda Pharmaceuticals
Brintellix (vortioxetine hydrobromide); NME
Tablet/oral
To treat adults with major depressive disorder

 
Takeda Pharmaceuticals
Nesina (alogliptin benzoate); NME
Tablet/oral
To improve blood sugar control in adults with Type 2 diabetes

 
Valeant Pharmaceuticals
Luzu (luliconazole); NME
Cream/topical
For the topical treatment of interdigital tinea pedis, tinea cruris, and tinea corporis caused by the organisms Trichophyton rubrum and Epidermophyton floccosum, in patients 18 years of age and older

 
ViiV Healthcare
Tivicay (dolutegravir sodium); NME
Tablet/oral
To treat HIV-1 infection
Fast Track; Priority Review
Approvals for new molecular entities (NMEs) filed under new drug applications and therapeutic biologics filed under original biologics license applications (BLAs) by FDA’s Center for Drug Evaluation and Research (CDER).

FDA Designations
Accelerated Approval: FDA’s Accelerated Approval program allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments. This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that FDA considers reasonably likely to predict clinical benefit. After this approval, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug.

Breakthrough Therapy: The Food and Drug Administration Safety and Innovation Act, passed in July 2012, gave the FDA the ability to designate a drug a breakthrough therapy at the request of the sponsor if preliminary clinical evidence indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. A breakthrough therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. 2013 was the first year any new drug was approved with the breakthrough designation.

Fast Track: Fast Track is given to drugs with the potential to address unmet medical needs. Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to developers and by enabling developers to use a “rolling review” process such that CDER can review portions of an application ahead of the submission of the full application.
           
Orphan Drugs: Orphan drugs are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment.

Priority Review: Priority Review is provided when FDA’s CDER determines the drug to potentially provide a significant advance in medical care and sets a target to review the drug within six months instead of the standard 10 months.

Other notes:
  • Bayer Healthcare formed an agreement in September 2009 with Algeta ASA (Oslo, Norway) for the development and commercialization of Xofigo.

  • In April 2014, GlaxoSmithKline agreed to sell its oncology portfolio to Novartis. The proposed transaction includes Mekinist and Tafinlar.

  • Janssen Biotech and Janssen Pharmaceuticals are part of Johnson & Johnson. Janssen Biotech and Pharmacyclics are partnered for Imbruvica. Janssen Pharmaceuticals has rights to Invokana through a license agreement with Mitsubishi Tanabe Pharma.

  • Roche Glycart AG, a wholly owned, independent research unit of Roche, discovered Gazyva. In the US, Gazyva is part of a collaboration between Genentech and Biogen Idec.

  • Roche licenses technology for Kadcyla under an agreement with ImmunoGen.

  • Sanofi’s subsidiary, Genzyme, partnered with ISIS Pharmaceuticals and Genzyme for the development of Kynamro.

  • Sunovion Pharmaceuticals is an indirect, wholly owned US subsidiary of Dainippon Sumitomo Pharma.

  • Takeda Pharmaceutical and H. Lundbeck A/S formed a strategic alliance for Brintellix in September 2007 under a broader alliance for several compounds in Lundbeck’s pipeline for the treatment of mood and anxiety disorders. The companies plan to copromote Brintellix in the US.

  • Valeant Pharmaceuticals acquired Medicis Pharmaceutical in 2012. Luzu (luliconazole) has been approved in Japan since 2005.

  • ViiV Healthcare is an independent pharmaceutical company focused on HIV established in 2009 by GlaxoSmithKline and Pfizer. Shionogi joined as a 10% shareholder in 2012.
Source: FDA
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