How did Phil Sharp, a kid from a small farm in Kentucky, become a Nobel prize winner and one of the godfathers of the biotech industry? How did Christine McSherry turn her youngest son’s devastating diagnosis into a crusade that aided in the approval of a drug for his disease? Sharp (pictured above) and McSherry … Continue reading “Catch Duchenne Patient Power, Phil Sharp & More Next Week at The Broad”
Author: Ben Fidler
Ben is former Xconomy Deputy Editor, Biotechnology. He is a seasoned business journalist that comes to Xconomy after a nine-year stint at The Deal, where he covered corporate transactions in industries ranging from biotech to auto parts and gaming. Most recently, Ben was The Deal’s senior healthcare writer, focusing on acquisitions, venture financings, IPOs, partnerships and industry trends in the pharmaceutical, biotech, diagnostics and med tech spaces. Ben wrote features on creative biotech financing models, analyses of middle market and large cap buyouts, spin-offs and restructurings, and enterprise pieces on legal issues such as pay-for-delay agreements and the Affordable Care Act. Before switching to the healthcare beat, Ben was The Deal's senior bankruptcy reporter, covering the restructurings of the Texas Rangers, Phoenix Coyotes, GM, Delphi, Trump Entertainment Resorts and Blockbuster, among others. Ben has a bachelor’s degree in English from Binghamton University.
Akcea, Fresh Off C-Suite Shakeup, Dishes Heart Drug to Pfizer for $250M
Pfizer this morning bought an experimental heart disease drug from Akcea therapeutics, the first move made by the Ionis Pharmaceuticals spinout since the company overhauled its management team last month. Pfizer will pay Akcea (NASDAQ: [[ticker:AKCA]]) and parent Ionis (NASDAQ: [[ticker:IONS]]) $250 million up front for rights to AKCEA-ANGPTL3-Lrx, an RNA-based medicine currently in a … Continue reading “Akcea, Fresh Off C-Suite Shakeup, Dishes Heart Drug to Pfizer for $250M”
Bio Roundup: PARP Progress, A New Commish, IPO-Palooza & More
It’s been a strange up and down ride for PARP inhibitors, a relatively new class of cancer drugs. Named for the tumor protein they target, PARPs rebounded after a big clinical setback in 2011 and have been at the center of many buyouts and big alliances—Pfizer and Medivation, GlaxoSmithKline and Tesaro, AstraZeneca and Merck. Four … Continue reading “Bio Roundup: PARP Progress, A New Commish, IPO-Palooza & More”
ESMO 2019: PARP and Prostate, SeaGen’s Win, KRAS Update & More
The European Society for Medical Oncology meeting has wrapped up in Barcelona, the last major clinical cancer conference until December’s annual ASH meeting for blood diseases. We’ve highlighted a few top stories for you. Amid all the fuss over cell, gene, and immunotherapies these days, an old-fashioned small-molecule class of drug called PARP inhibitors have … Continue reading “ESMO 2019: PARP and Prostate, SeaGen’s Win, KRAS Update & More”
Enanta Steps Forward in NASH Race, With Unclear Prospects
Enanta Pharmaceuticals late Wednesday became the latest of a number of companies to tout an emerging potential therapy for nonalcoholic steatohepatitis, the fatty liver disease that is fast becoming a global epidemic. But it’s unclear whether the experimental treatment can stand out from the crowd. Enanta (NASDAQ: [[ticker:ENTA]]) said the higher of two tested doses of … Continue reading “Enanta Steps Forward in NASH Race, With Unclear Prospects”
MedCo Preps for FDA Filing as Cholesterol Drug Clears Last Two Tests
The Medicines Co. this morning inched closer to bringing a twice-yearly cholesterol-lowering drug to market, touting a positive result in two more Phase 3 studies. Without providing details, MedCo (NASDAQ: [[ticker:MDCO]]) said that the experimental medicine, inclisiran, hit all of its main and secondary goals in two Phase 3 studies called ORION-9 and ORION-10. Like … Continue reading “MedCo Preps for FDA Filing as Cholesterol Drug Clears Last Two Tests”
Flagship’s Omega Aims to Take Epigenetic Drugs a Step Further
A biotech startup called Omega Therapeutics debuts this morning touting a new approach to developing medicines based on epigenetics, a field of research that has tantalized drug makers for years but produced mixed results. Can the fledgling company surpass previous efforts and develop epigenetic drugs for a slew of different diseases? Cambridge, MA-based Omega was … Continue reading “Flagship’s Omega Aims to Take Epigenetic Drugs a Step Further”
Bio Roundup: Award Winners, Lung Data, Dems on Drug Pricing & More
For decades, advanced lung cancer was a quick death sentence. That’s no longer a given, thanks in part to the arrival of immunotherapy. If a medical meeting in Barcelona this week was any indication, more help could be on the way. Targeted medicine isn’t new to lung cancer, but only a fraction of patients have … Continue reading “Bio Roundup: Award Winners, Lung Data, Dems on Drug Pricing & More”
Phil Sharp, CRISPR’ing the Heart & the Duchenne Data Gap on Oct. 17
Phil Sharp is one of the godfathers of biotech. He’s an MIT biologist, Nobel laureate and, as a Biogen (NASDAQ: [[ticker:BIIB]]) co-founder, one of the people responsible for turning Kendall Square into the biopharma epicenter it is today. Next month, he will share his story, the lessons he’s learned along the way, what he’s up … Continue reading “Phil Sharp, CRISPR’ing the Heart & the Duchenne Data Gap on Oct. 17”
Herceptin Inventors, Immunology Pioneers Take Home 2019 Lasker Awards
This year’s Lasker Awards, the US’s most prestigious biomedical honor, are going to five scientists whose work led to a critical breast cancer treatment and significant basic research advances that have helped pave the way for immunotherapy. H. Michael Shepard, Dennis Slamon, and Axel Ullrich won the Lasker-DeBakey Clinical Medical Research Award for inventing trastuzumab … Continue reading “Herceptin Inventors, Immunology Pioneers Take Home 2019 Lasker Awards”
As New CEO of Cerevel, Coles Moves From One Neuro Startup to Another
Tony Coles, the biotech veteran who once steered Onyx Pharmaceuticals into a $10 billion buyout, has left one neurology startup to join another. Coles on Monday was named the CEO of Cerevel Therapeutics, a Boston company that Pfizer (NYSE: [[ticker:PFE]]) and Bain Capital launched in October 2018 with $350 million in funding. The appointment marks the … Continue reading “As New CEO of Cerevel, Coles Moves From One Neuro Startup to Another”
At Big Lung Cancer Meeting, Lights Shine on KRAS, Drug Combos & More
The treatment landscape for lung cancer has shifted significantly over the past few years, and more changes could be on the way. At the World Conference on Lung Cancer in Barcelona this weekend a number of drug makers trotted out some of their latest advances in immunotherapy, targeted pills, drug combinations, and more. Xconomy rounded … Continue reading “At Big Lung Cancer Meeting, Lights Shine on KRAS, Drug Combos & More”
With FDA’s Eye on JAK Drugs, Concert Moves One Ahead For Hair Loss
Despite the safety concerns that have recently emerged for class of drugs known as JAK inhibitors, their reach continues to grow. The latest example comes from Concert Pharmaceuticals, which is racing the drug giant Pfizer to develop a medicine for a common skin disease that causes hair loss. Concert (NASDAQ: [[ticker:CNCE]]) is disclosing this morning … Continue reading “With FDA’s Eye on JAK Drugs, Concert Moves One Ahead For Hair Loss”
As Cholesterol Drug Aces Big Test, MedCo CEO Open to Flexible Pricing
The Medicines Co. (NASDAQ: [[ticker:MDCO]]) announced last week that its cholesterol-lowering medicine inclisiran, meant to be taken just twice a year, had passed a key test but offered no details. Some of those details arrived this morning. At a medical meeting in Paris, a presentation from the test, a 1,617-patient Phase 3 study called ORION-11, … Continue reading “As Cholesterol Drug Aces Big Test, MedCo CEO Open to Flexible Pricing”
$5.8B for Nothing: AbbVie Shelves Stemcentrx Drug After Latest Flop
AbbVie has officially waved the white flag on the cancer drug that triggered its $5.8 billion buyout of Stemcentrx a few years ago. North Chicago, IL-based AbbVie (NYSE: [[ticker:ABBV]]) said that rovalpituzumab tesirine, or Rova-T, failed another clinical trial—this time a Phase 3 study, MERU, testing the drug as a maintenance therapy in patients with … Continue reading “$5.8B for Nothing: AbbVie Shelves Stemcentrx Drug After Latest Flop”
Celgene Adds Cell Therapies From Immatics With Bristol Sale in Sight
Even as its sale to Bristol-Myers Squibb (NYSE: [[ticker:BMY]]) inches closer to completion, Celgene—long known for its web of biotech partnerships—has inked another alliance. Celgene (NASDAQ: [[ticker:CELG]]) will co-develop three cell therapies for cancer with Immatics, a German biotech with ties to MD Anderson Center Center in Houston. Celgene will pay Immatics $75 million in … Continue reading “Celgene Adds Cell Therapies From Immatics With Bristol Sale in Sight”
Retrophin Sinks as Drug “Pharma Bro” Martin Shkreli Co-Invented Fails
[Corrected, 8/23/19, see below] Retrophin, the drug developer founded by jailed former biotech executive Martin Shkreli, has failed its first big test. The San Diego-based company’s experimental drug fosmetpantotenate failed a Phase 3 trial, FORT, in patients with a rare and deadly neurological disorder called pantothenate kinase-associated neurodegeneration, or PKAN. The Retrophin (NASDAQ: [[ticker:RTRX]]) drug … Continue reading “Retrophin Sinks as Drug “Pharma Bro” Martin Shkreli Co-Invented Fails”
FDA Rejects Sarepta’s Second Duchenne Drug, Citing Safety Concerns
[Updated, 8/20/19, see below] Sarepta Therapeutics won one of the most dramatic and controversial drug approvals in the history of the FDA a few years ago. It didn’t have the same luck the second time around. The FDA late Monday rejected golodirsen (Vyondys 53), which was widely expected to become the second approved Duchenne muscular … Continue reading “FDA Rejects Sarepta’s Second Duchenne Drug, Citing Safety Concerns”
Four New Drugs Are Around the Corner. Here’s What You Need to Know.
[Updated, 3:40 pm ET, see below] The Food and Drug Administration approved 59 new drugs last year, a record for the agency which over the years has swung back and forth between tight control and leniency. We are in the midst of perhaps the agency’s most permissive era ever, thanks to its openness to speed … Continue reading “Four New Drugs Are Around the Corner. Here’s What You Need to Know.”
Chasing Blueprint, Deciphera Plans FDA Filing for GI Cancer Drug
[Updated, 11:04 am ET, see below] Deciphera Pharmaceuticals this morning announced plans to seek FDA approval of a cancer medicine that treats a deadly type of tumor that forms in the gastrointestinal tract. The Deciphera (NASDASQ: [[ticker:DCPH]]) drug, ripretinib, hit its main goal in a Phase 3 trial, INVICTUS, in 129 patients with gastrointestinal stromal … Continue reading “Chasing Blueprint, Deciphera Plans FDA Filing for GI Cancer Drug”
Bio Roundup: Surprise News, a Gene Therapy Mess & a CAR-T Step
Gene therapy has become one of the hottest fields in biomedicine, with two treatments approved in the US already and several more on the way. But surprise news this week regarding Zolgensma, the recently approved spinal muscular atrophy treatment, cast a cloud over its progress. The FDA revealed that Novartis subsidiary AveXis had “manipulated” data … Continue reading “Bio Roundup: Surprise News, a Gene Therapy Mess & a CAR-T Step”
Bayer Buys Out BlueRock, Betting at Least $240M More on Cell Therapy Work
Versant Ventures and Bayer have started several biotechs together. But today marks the first time the German drug maker takes a step further and buys one of them: cell therapy developer BlueRock Therapeutics. Bayer already owns a 40.8 percent stake in BlueRock through its Leaps by Bayer venture arm. But it will pay $240 million … Continue reading “Bayer Buys Out BlueRock, Betting at Least $240M More on Cell Therapy Work”
FDA: Despite “Manipulated” Data, $2M Gene Therapy Should Stay on Market
[Updated, 7:12 pm ET, see below] The FDA on Tuesday said that some data supporting the spinal muscular atrophy gene therapy Zolgensma was “manipulated” before its May 24 approval and that its manufacturer, Novartis (NYSE: [[ticker:NVS]]) subsidiary AveXis, knew about it. The FDA stopped short of saying Zolgensma should be pulled from the market, but … Continue reading “FDA: Despite “Manipulated” Data, $2M Gene Therapy Should Stay on Market”
GlycoMimetics Sickle Cell Drug Flops as Others Creep Forward
New treatments could soon be on the way for sickle cell disease, an inherited and debilitating blood disorder. But a drug from GlycoMimetics won’t be among them. GlycoMimetics (NASDAQ: [[ticker:GLYC]]) had been aiming to show that a drug called rivipansel could help sickle cell patients who are hospitalized with severe pain episodes, or “crises.” The … Continue reading “GlycoMimetics Sickle Cell Drug Flops as Others Creep Forward”
Amicus Offers First Look At Gene Therapy Work With Batten’s Data
Amicus Therapeutics bought a spinout of Nationwide Children’s Hospital last year as part of a plan to become a player in the emerging field of gene therapy. Today it is providing the first evidence, in humans, that the investment could pay dividends. The results Amicus (NASDAQ: [[ticker:FOLD]]) are disclosing this morning come with plenty of … Continue reading “Amicus Offers First Look At Gene Therapy Work With Batten’s Data”
Merck Data Are Another Step for Immunotherapy in Breast Cancer
Merck this morning announced that a combination of its drug pembrolizumab (Keytruda) and chemotherapy succeeded in a Phase 3 study in breast cancer, a potentially notable advance for immunotherapy in treating the disease. Merck (NYSE: [[ticker:MRK]]) said that Keytruda and chemotherapy beat chemo alone when given to patients with triple-negative breast cancer in a study … Continue reading “Merck Data Are Another Step for Immunotherapy in Breast Cancer”
Duchenne Advocates Start Casimir, Aiming to Capture Missing Data
Three years ago, the FDA made one of the most polarizing decisions in its history. It approved a drug for the rare genetic disease Duchenne muscular dystrophy on the slimmest of evidence, a watershed moment that caused a highly publicized rift within the agency. In the midst of it all were parents who pushed hard … Continue reading “Duchenne Advocates Start Casimir, Aiming to Capture Missing Data”
Bio Roundup: Leiden’s Exit, Depression Data, a New Pricing Bill & More
What will the legacy be of Jeff Leiden, the longtime CEO of Vertex Pharmaceuticals? Vertex surprised Wall Street this week by announcing Leiden’s seven-year run will end next spring. In some ways, his legacy is already written. He steered Vertex through a crisis when its hepatitis C business was squashed by competition, in no small … Continue reading “Bio Roundup: Leiden’s Exit, Depression Data, a New Pricing Bill & More”
Jeff Leiden, Who Shaped Vertex Into a CF Force, to Switch Roles
Jeff Leiden, who helped steer Vertex Pharmaceuticals out of a crisis and into a profitable developer of cystic fibrosis medicines, will step down as the CEO of the Boston company next spring. In a surprising announcement, Vertex (NASDAQ: [[ticker:VRTX]]) said Thursday morning that Leiden (pictured), who has run the company for about seven years, will … Continue reading “Jeff Leiden, Who Shaped Vertex Into a CF Force, to Switch Roles”
Bristol, With Another Lung Cancer Flop, Loses More Ground to Merck
Bristol-Myers Squibb (NYSE: [[ticker:BMY]]) this afternoon revealed yet another setback for its cancer immunotherapy nivolumab (Opdivo) in lung cancer, boosting the outlook for rival Merck. The New York pharma giant said that a regimen of nivolumab (Opdivo) and chemotherapy failed to extend the lives of non-squamous, non-small cell lung cancer patients compared to chemo alone … Continue reading “Bristol, With Another Lung Cancer Flop, Loses More Ground to Merck”
Gilead Ups Stake in Galapagos With $5B Deal to Jumpstart Its R&D
Gilead Sciences on Sunday looked to another firm to rejuvenate its research capabilities, agreeing to put more than $5 billion in cash into Galapagos NV and get access to a slew of experimental drugs in return. Gilead (NASDAQ: [[ticker:GILD]]) will pay Galapagos (NASDAQ: [[ticker:GLPG]]) a whopping $3.95 billion in cash and make a $1.1 billion … Continue reading “Gilead Ups Stake in Galapagos With $5B Deal to Jumpstart Its R&D”
Neon’s Early Vaccine Study Is a Peek at Immunotherapy’s Third Wave
In this new age of cancer immunotherapy, two versions have been approved. The first are checkpoint inhibitors, which have begun to change the way skin, lung, and other cancers are treated. The second are CAR-T cell therapies, which have shown promise in blood cancers. A third type of cancer immunotherapy is just now reaching clinical … Continue reading “Neon’s Early Vaccine Study Is a Peek at Immunotherapy’s Third Wave”
As Sangamo Touts Data, BioMarin Preps Hemophilia Gene Therapy For FDA
[Updated 7/8/19, 9:22 am. See below.] Gene therapy for hemophilia is as close as it’s ever been to market. And the race to get there got more heated this past weekend at a medical meeting in Australia. At the International Society on Thrombosis and Haemostasis meeting in Melbourne, Sangamo Therapeutics (NASDAQ: [[ticker:SGMO]]) and partner Pfizer … Continue reading “As Sangamo Touts Data, BioMarin Preps Hemophilia Gene Therapy For FDA”
Bio Roundup: Duchenne Race, Warren v. Gottlieb, A $599 Genome & More
As we pause to celebrate the 4th of July, another birthday is top of mind: A third baby whose embryonic DNA was edited by Chinese scientist He Jiankui. As Xconomy reported in November, He hinted about a second pregnancy after his infamous revelation of twins altered with CRISPR gene editing tools. That second pregnancy—a third … Continue reading “Bio Roundup: Duchenne Race, Warren v. Gottlieb, A $599 Genome & More”
Encoded Nabs $104M, Illumina’s Help, to Push Gene Therapy’s Limits
Despite the progress of gene therapy—a cutting edge medicine promising long-lasting effects from a single treatment—it remains a crude and limited tool. Startup Encoded Therapeutics has raised $104 million to join the race to expand gene therapy’s reach. The South San Francisco, CA, company has emerged from the startup accelerator of Illumina (NASDAQ: [[ticker:ILMN]]), and … Continue reading “Encoded Nabs $104M, Illumina’s Help, to Push Gene Therapy’s Limits”
AbbVie to Pay $63B for Allergan to Prepare for Life Without Humira
[Updated, 10:11 am ET, see below.] Pharmaceutical giant AbbVie this morning agreed to acquire Allergan in a $63 billion deal meant to provide the pharmaceutical giant with enough revenue to brace for the loss of patent protection for the world’s top-selling drug. AbbVie (NYSE: [[ticker:ABBV]]) will pay $188.24 per share in cash and stock for … Continue reading “AbbVie to Pay $63B for Allergan to Prepare for Life Without Humira”
Colorectal Cancer Data Spurs Pfizer to Pay $11.4B for Array Bio
[Updated 11:02 a.m. See below.] Array BioPharma touted data less than a month ago that could shift the treatment landscape for some patients with metastatic colorectal cancer. The data caught the eye of Pfizer, which just agreed this morning to buy the Boulder, CO, cancer drug maker for $11.4 billion. Pfizer (NYSE: [[ticker:PFE]]) will pay … Continue reading “Colorectal Cancer Data Spurs Pfizer to Pay $11.4B for Array Bio”
Bio Roundup: Roche-Spark Drags On, GSK Taps CRISPR, Diabetes News & More
Is one of the bigger biopharma acquisitions of the year in trouble? This past week, antitrust regulators once again delayed Roche’s planned $4.8 billion buyout of gene therapy developer Spark Therapeutics (NASDAQ: [[ticker:ONCE]]). The US Federal Trade Commission wants yet more information about the buyout, and overseas, the UK Competition and Markets Authority opened a … Continue reading “Bio Roundup: Roche-Spark Drags On, GSK Taps CRISPR, Diabetes News & More”
Can AI Tools, $76M Lead BlackThorn to Targeted Psych Drugs?
It’s notoriously tough for experimental psychiatric drugs to succeed in clinical tests. BlackThorn Therapeutics wants to show that an AI-driven, precision approach can make it easier, and today it’s added another $76 million to get the chance to prove it. The new Series B round will give San Francisco-based BlackThorn—a startup spun out of the … Continue reading “Can AI Tools, $76M Lead BlackThorn to Targeted Psych Drugs?”
CymaBay Therapeutics Stumbles in NASH Race, Shares Fall
One of the entrants in the ultra-competitive race to develop treatments for the fatty liver disease known nonalcoholic steatohepatitis (NASH) suffered a setback in clinical testing today, but the company, Cymabay Therapeutics, still believes its drug could show promise as the study continues. Cymabay (NASDAQ: [[ticker:CBAY]]), of Newark, CA, reported interim results from a Phase … Continue reading “CymaBay Therapeutics Stumbles in NASH Race, Shares Fall”
Merck Bags Tilos, Adding an Immunotherapy Booster to Its Arsenal
Cancer immunotherapy leader Merck continued to roll up oncology assets with a deal this morning to acquire privately held Tilos Therapeutics. Merck (NYSE: [[ticker:MRK]]) didn’t disclose how much it will pay up front for Tilos, a Lexington, MA, startup developing drugs for cancer, fibrosis, and autoimmune diseases. But the overall payout could reach $773 million … Continue reading “Merck Bags Tilos, Adding an Immunotherapy Booster to Its Arsenal”
Immunotherapy Is Now Here For Breast Cancer. What Are Its Prospects?
Drugs that rev up a patient’s immune system have changed how we treat cancers of the skin, lung, and more, offering people whose tumors have spread a chance to live longer than ever thought possible. But until this year, the field had nothing for breast cancer, which kills more women than any other cancer type. … Continue reading “Immunotherapy Is Now Here For Breast Cancer. What Are Its Prospects?”
Second US Gene Therapy, Approved for Rare Muscle Disease, to Cost $2M
The FDA today has made Zolgensma, a Novartis treatment for the rare genetic disease spinal muscular atrophy, the second approved gene therapy in the US. The news marks a milestone for SMA patients, who have only one other approved medicine available. But it comes with a cost: At $2.125 million, Zolgensma’s price tag creates a … Continue reading “Second US Gene Therapy, Approved for Rare Muscle Disease, to Cost $2M”
MedCo Touts New Data for Long-Lasting Heart Drug as Reckoning Looms
The Medicines Co. and partner Alnylam Pharmaceuticals have presented new evidence supporting the long-term use of their RNA-based cholesterol lowering therapy inclisiran, which is meant to be a longer lasting alternative to a new crop of heart drugs called PCSK9 inhibitors. At a medical meeting in Miami, Medicines Co. (NASDAQ: [[ticker:MDCO]]) reported that 290 patients … Continue reading “MedCo Touts New Data for Long-Lasting Heart Drug as Reckoning Looms”
ASCO Abstracts: Breast Cancer, Precision Treatments in the Spotlight
The world’s biggest annual cancer conference is just around the corner. Data presented at the American Society of Clinical Oncology meeting each year can change medical practice and make or break companies whose drugs are under the microscope. That’s what makes even the “abstracts,” or snippets of data revealed a few weeks before the meeting, … Continue reading “ASCO Abstracts: Breast Cancer, Precision Treatments in the Spotlight”
Vertex Continues Plan to Diversify, Strikes a $70M Pact With Kymera
Vertex Pharmaceuticals is best known for its cystic fibrosis franchise. But over the past several years Vertex has been making inroads elsewhere, and a deal it is announcing this morning continues that strategy. Vertex (NASDAQ: [[ticker:VRTX]]), of Boston, will pay privately held startup Kymera Therapeutics $70 million to form an alliance to develop a group … Continue reading “Vertex Continues Plan to Diversify, Strikes a $70M Pact With Kymera”
Bio Roundup: Rare Disease Showdowns, Prices on TV, Dealmania & More
The market for drugs for rare or “orphan” diseases continues to grow. An April report from Evaluate Pharma predicted sales of orphan drugs to rise 12 percent a year between now and 2024, when they will account for an estimated 20 percent of worldwide prescription drug sales. The field has gotten so competitive that multiple … Continue reading “Bio Roundup: Rare Disease Showdowns, Prices on TV, Dealmania & More”
SMA, Migraine Data Lead Neuro Highlights from AAN Meeting
The American Academy of Neurology meeting in Philadelphia is rolling on through the end of this week. There has been plenty of news to digest already. New clinical data could herald cutting-edge treatments for migraine, the rare disease spinal muscular atrophy, the deadly neurological disorder amyotrophic lateral sclerosis, and more. Xconomy previewed the conference last … Continue reading “SMA, Migraine Data Lead Neuro Highlights from AAN Meeting”
FDA OKs Pfizer Rare Disease Drug, Sets Up Battle With Alnylam, Akcea
[Updated 5/6/19, 9:20 am ET. See below.] A year ago, there were no FDA-approved medicines available for US patients with the debilitating rare genetic disease transthyretin amyloidosis (ATTR). Things have changed fast, however: With the FDA’s decision to approve a drug from Pfizer this morning, patients now have three options to choose from. The FDA … Continue reading “FDA OKs Pfizer Rare Disease Drug, Sets Up Battle With Alnylam, Akcea”
SMA Moment: Will Gene Therapy Shift Treatment, Costs of Muscle Disease?
This is a big moment for people diagnosed with spinal muscular atrophy, or SMA, a rare and potentially lethal genetic disorder that destroys muscles. For decades, there was no way to change the trajectory of their disease. They now have one marketed medicine, and this month, chances are they’ll have another: a gene therapy that … Continue reading “SMA Moment: Will Gene Therapy Shift Treatment, Costs of Muscle Disease?”