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

$8.7 Billion SMA Drug Explained

Emily BurkeGene Therapy, Orphan Drugs, SMA

The Science Behind the Deal Earlier this week, news of the $8.7 billion acquisition of gene therapy company AveXis (Bannockburn, IL) by Novartis (Basel, Switzerland) made big biotech headlines. AveXis’ lead candidate, AVXS-101, is now in Phase III clinical studies for the treatment of spinal muscular atrophy (SMA). In this Weekly, we’ll take a look at the science behind these headlines by explaining exactly … 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

Nanobodies: These Are Not Your Mother’s mAbs

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs

The Drug Kingpins Monoclonal antibodies (mAbs) are the undisputed drug kingpins. In 2013, the mAb market raked in $75 billion in combined sales, covering a whole range of indications from cancer and infectious disease, to autoimmune disorders, and even high cholesterol. Despite the success, mAbs have one chink in their armor: they cannot enter cells due to their large size, hampering their range … Read More

RNA Therapeutics March Onward

Emily BurkeAntisense, Biotech Basics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

Taking Steps With Antisense With their high specificity and relative low manufacturing cost, RNA therapeutics may be tomorrow’s biotech sweetheart. In fact, chances are good that previously “undruggable” targets that cannot be accessed by small or large molecule drugs, are now within reach. However, the main roadblock continues to be delivery—getting the RNA drug where it needs to be, in high enough concentrations, to … Read More

New Hope For Spinal Muscular Atrophy

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

FIRST THERAPY APPROVED FOR SMA Squeaking by on December 23rd as the last new drug approval of 2016, Biogen’s (Cambridge, MA) Spinraza now provides hope for the thousands of families affected by a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA). SMA robs people of their ability to walk, eat, and ultimately, breathe. In addition to Spinraza, there are 13 … Read More

The Race To Beat SMA

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

Zeroing In On The SMA Pipeline A decade ago, there was only one drug in development for a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA) — robbing people of their ability to walk, eat, and ultimately, breathe. Today, there are 14 therapies making their way through the clinic according to the patient advocacy group Cure SMA. The increase is … 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

A Drop Of Gene Therapy

Emily BurkeBiologics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, The WEEKLY

A Walk In The Park For Hemophilia? Imagine tripping over your feet during a leisurely stroll down the sidewalk. Ouch! Your knees are scraped below your shorts and blood starts to drip. A quick wipe from a conveniently pocketed napkin and soon enough, you are the proud owner of some new scabs. Life goes on. If you are one of … Read More

On The Hunt

Emily BurkeBiotech Basics, Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Term of the Week, The WEEKLY

The Hurdles Of Huntington’s The nervous system is an incredibly complex piece of human machinery, stretching to the far reaches of the body while controlling and receiving the nuances of life from a central command station. Just like any part of the human body, the central nervous system (CNS) is affected by various diseases that are sometimes not entirely understood. After … 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

Deciphering DMD

Emily BurkeAntisense, Clinical Trials, Drug Development, Drug Targets, FDA, Genetics, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), Small Molecule Drugs

THE ROOT OF DUCHENNE MUSCULAR DYSTROPHY World Duchenne Awareness Day called attention to Duchenne Muscular Dystrophy (DMD) this past Monday. Affecting one in 3,500 newborn baby boys worldwide, this fatal disease is caused by a mutation in the gene that codes for the dystrophin protein. Easily damaged muscle cells, progressive muscular weakness, and serious medical problems—including significant impairment of the heart and lungs—are the direct results of this … Read More

A Drop Of Biotech

Emily BurkeCancer, Clinical Trials, Cocktail Fodder, Diagnostics, Drug Development, Genomics, HIV, Orphan Disease, Orphan Drugs, The WEEKLY

Basics of Blood Disorders Blood is the carrier of a multitude of fundamental body processes—responsible for delivering vital nutrients/oxygen and for removing wastes. Like the highway exchange feeding a city of life, blood is simply essential. The branch of medicine concerned with the study, diagnosis, treatment, and prevention of blood related diseases is known as hematology. This single therapeutic area covers a broad range … Read More

Targeting The Cause Of Cystic Fibrosis

Emily BurkeBiologics, Clinical Trials, Drug Approvals, Drug Development, Drug Targets, FDA, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

Biotech Changes The Game The FDA approval of Vertex Pharmaceutical’s (Boston, MA) Orkambi last week further shifted the paradigm of treating cystic fibrosis (CF) for up to half of its sufferers—moving from a management of symptoms approach to targeting the underlying cause. Until very recently, the only strategy against CF involved reducing the risk of lung infections by taking mucus thinning medications and antibiotics—and rarely, late stage bilateral lung transplants. CF … Read More

The State Of Cystic Fibrosis And Precision Medicine

Emily BurkeCancer, Clinical Trials, Drug Approvals, Drug Development, Drug Targets, Easily Confused, FDA, Genetics, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

During President Obama’s State of the Union address last month, a cystic fibrosis patient named Bill Elder sat beside First Lady Michelle Obama. Diagnosed with the disease at 8 years old, Mr. Elder is “healthier now than ever before” at age 27, thanks to Vertex’s (Boston, MA) Kalydeco. As a third-year medical student, he is not only surviving, but thriving. Receiving an invitation to … Read More