Rare Disease Focus: PKU

Emily BurkeDrug Approvals, Drug Development, Edited by Sarah Van Tiem, Orphan Disease, Orphan Drugs

Biopharma To The Rescue: PKU The ubiquitous soda can. Who hasn’t seen one? Ever look on the back, at the disturbingly long paragraph of ingredients? The list of ingredients on the back of a can of diet soda are perhaps even more unsettling. Underneath it, there’s a warning in bold: “Phenylketonurics: Contains Phenylalanine.” Phenylalanine doesn’t harm most people. But what’s … Read More

Groundbreaking Migraine Drug Explained

Emily BurkeAuthor Emily Burke PhD, Drug Approvals, Drug Development, Drug Discovery, Drug Targets, Edited by Sarah Van Tiem, Migraine

FIRST IN CLASS MIGRAINE APPROVAL Last month the FDA approved Amgen’s (Thousand Oaks, CA) new migraine drug Aimovig, the first drug shown to prevent the onset of migraines. The drug significantly reduces the number of migraine days in difficult-to-treat (those that have failed 2 to 4 prior treatments) patient populations. In some patients dubbed “super responders”, migraines occurrence went from several times/month to no occurrence for 6 … Read More

From Drug Development to Approval: A Recap

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Discovery, Drug Targets, FDA

From The Lab To The Patient In this issue of the Biotech Primer WEEKLY we will recap the past seven issues that highlight the journey a molecule takes from the lab to the patient. Beginning in the 1980’s, scientists took a new tack in developing drugs. They adopted an approach known as rational drug discovery. Using this methodology, researchers first … Read More

Market Access

Linda LanderDrug Approvals, Market Access, The WEEKLY

Market Access Primer  For the last few weeks we here at Biotech Primer have tracked the progression of a drug candidate from the lab to the marketplace, where only the fittest survive. Winning at clinical trials means earning an official regulatory approval. Congratulations! But as any seasoned drug developer will tell you, the game has only just begun. Ensuring newly-approved … Read More

From Drug Development To Approval: Phase IV

Emily BurkeClinical Trials, Cocktail Fodder, Drug Approvals, Drug Development, FDA, Orphan Drugs

Pharma Finish Line: FDA Approval Last week, we focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of truth: does the study data support claims that the new drug is both safe … Read More

From Drug Development To Approval: Phase III

Emily BurkeAuthor Emily Burke PhD, Drug Approvals, Drug Development, FDA, The WEEKLY

Phase III Is No Guarantee Our last Biotech Primer WEEKLY explored the riskiest part of the human clinical trials pathway: Phase II. About 70% of drugs that enter Phase II never make it out. Most often, it’s because they fail to demonstrate effectiveness. Even making it to Phase III is no guarantee of success – about 40% of drugs fizzle … Read More

Stopping A Big Problem: Blood Clots

Emily BurkeCocktail Fodder, Drug Approvals, Drug Targets, Mechanism of Action, Small Molecule Drugs

MEDS FOR THINNER BLOOD CAN EQUAL FEWER CLOTS, BUT HOW? The FDA’s recent approval of Portola Pharmaceuticals’ (South San Francisco, CA) new blood thinner drug Bevyxxa paved the way for the prevention of blood clots in patients hospitalized for conditions such as heart failure, stroke, and pulmonary disease. The medical term for blood clot is venous thromboembolism (VTE), but if we take it apart: “venous” means relating … Read More

Decoding Your Genes

Emily BurkeBiotech Basics, Deoxyribonucleic Acid (DNA), Diagnostics, Drug Approvals, Easily Confused, FDA, Genetics, Genomics, Orphan Disease, Term of the Week, The WEEKLY

The Skinny On DNA Testing 23andMe (Mountain View, CA) recently found itself back in the limelight after the disease risk section of its mail-in DNA kit received an OK from the FDA. The Silicon Valley biotech had to halt sales of its direct-to-consumer genetics testing back in 2013 after regulatory officials grew concerned over marketing claims and the possibility of consumers misinterpreting the test results. 23andMe rebooted a limited part of … Read More

The PARP Race Is On

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, Easily Confused, FDA, Mechanism of Action, The WEEKLY

PARP1 INHIBITOR LINEUP PARP1 inhibitors are making a strong statement! Tesaro’s (Waltham, MA) just-approved Zejula has garnered predictions of blockbuster status. AstraZeneca’s (Cambridge, UK) Lynparza was the first PARP1 inhibitor to make it to market back in 2014, and their recent clinical trial results showed significant survival benefit in ovarian cancer. Clovis Oncology (Boulder, CO) achieved the second FDA approval of a PARP1 inhibitor with Rubraca in … Read More

DMD Makes The Cut

Emily BurkeAntisense, Biologics, Clinical Trials, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, FDA, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

The Science Behind Sarepta’s Hotly Debated Antisense Drug Sarepta’s (Cambridge, MA) Duchenne muscular dystrophy drug Exondys 51 crossed the finish line earlier this week, with a conditional stamp of approval by the FDA. This hotly debated regulatory result offers new hope for patients and families whose previous treatment options only managed the descent of the disease. Affecting one in approximately 3,500 … Read More

The Next Generation Fight Against CF

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Targets, Easily Confused, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, The WEEKLY

TARGETING THE ROOT OF CYSTIC FIBROSIS Innovative therapies targeting the root cause of cystic fibrosis (CF) hit the market several years ago, but those treatments were only for a subset of CF patients.  Now, companies like AbbVie and Vertex may have the potential to treat a large majority of the population—up to 90%—according to clinical trial data. In this issue, we’ll explain … Read More

The Antibiotic Economy

Emily BurkeBiotech Basics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, The WEEKLY

THE STATE OF ANTIBACTERIALS One of the greatest public health challenges of the 21st century is antibiotic resistance, which occurs when a few bacteria in a given population develop a genetic mutation that enables them to survive—even in the presence of antibiotics. How do bacteria become drug resistant? Suppose a particular antibiotic inhibits an enzyme required for bacterial replication. If one bacterium … Read More

Pushing The Self-Destruct Button

Emily BurkeCancer, Drug Approvals, FDA, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

 Pushing the Self-Destruct Button Breakthrough drug Venclexta charged onto the marketplace three months early to battle chronic lymphocytic leukemia. AbbVie’s (North Chicago, IL) and Roche’s (Basel, Switzerland) new therapy gained a quick approval after 80% of patients in the 106-person clinical trial responded to the small molecule inhibitor. Chronic lymphocytic leukemia (CLL) is the most common type of leukemia in adults, with approximately … Read More

Two Monoclonal Antibodies Walk Into The Market

Emily BurkeBiologics, Clinical Trials, Drug Approvals, Drug Development, Drug Targets, Easily Confused, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, Term of the Week, The WEEKLY

Attacking Asthma & Pushing Out Psoriasis Two new monoclonal antibodies debuted on the market thanks to FDA approvals last month. Both treat chronic inflammatory conditions: Lilly’s (Indianapolis, IN) Taltz for psoriasis and Teva’s (Petah Tikva, Israel) drug Cinqair for severe asthma. Interestingly enough, having psoriasis is associated with an increased risk of developing asthma according to a study in the British Journal of Dermatology. While these two conditions … Read More

Orphan Drugs Lead 2015 Approvals

Emily BurkeBiologics, Cardiovascular Disease, Drug Approvals, FDA, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

Treatment Options Broaden Driven by 19 new orphan approvals, a whopping 45 novel drugs were cleared for the marketplace in 2015. Additionally, new approvals opened up treatment options for Americans with diseases ranging from breast and lung cancers to irritable bowel syndrome. According to FDA numbers: 19 were for orphan diseases that affect fewer than 200,000 patients per year in the US. … Read More

Cancer Immunotherapy Goes Viral

Emily BurkeBiologics, Cancer, Drug Approvals, Drug Targets, FDA, Mechanism of Action, The WEEKLY

Oncolytic Viruses Make Their Debut Does a virus engineered to harness the immune system to fight cancer sound like a clever idea? Amgen (Thousand Oaks, CA) certainly thinks so, because their talimogene laherparepvec (T-Vec) recently earned an FDA approval to fight inoperable melanoma recurrent after initial surgery. Oncolytic viruses—like T-Vec—have the attention of both industry media and mainstream news programs. This new class of therapy is an elegant “hack” of the immune … Read More