A decade later, biotech’s CRISPR revolution is still going strong

Once the specialty of a few select drugmakers, CRISPR gene editing is now an essential technology for a growing group of biotechs, many led by former students of the field's pioneering scientists.

By Gwendolyn Wu , Shaun Lucas , Julia Himmel • Oct. 12, 2023

FDA’s ‘ivory tower thinking’ ignores promising biomarkers, says a rare disease CEO

The FDA wouldn’t review a drug that was 90% effective. In rare diseases, this is too common, said Ultragenyx CEO Dr. Emil Kakkis and a leading rare disease researcher.

By Michael Gibney • Oct. 5, 2023

Explore the Trendline➔

Rare diseases

As drug developers find promise in the rare diseases, deals and treatments are on the rise

By PharmaVoice staff

Drugmakers face uphill battle in Medicare negotiation suits

Judges have found fault with drug manufacturers’ First and Fifth Amendment arguments against the IRA’s drug pricing measures.

By Karissa Waddick • Oct. 4, 2023

5 FDA decisions to watch in the fourth quarter

The regulator is considering approval of what would be the first CRISPR medicine, as well as important clearances for Alnylam, Bristol Myers, Amgen and Pfizer.

By Ned Pagliarulo , Jonathan Gardner • Oct. 2, 2023

3 recent FDA adcomm rejections — and why they matter

Decisions that could spell doom for drugs in development and already on the shelves.

By Meagan Parrish • Sept. 29, 2023

How pharma marketers can reclaim the drug pricing narrative

As Medicare negotiations heat up, it’s more important than ever for pharma companies to communicate their value. Here’s how.

By Karissa Waddick • Sept. 28, 2023

As the opioid crisis seethes, a long-lasting treatment could offer new ammunition

Launched this month, Brixadi from Braeburn gives patients a new tool to overcome addiction.

By Michael Gibney • Sept. 21, 2023

PBMs, PhRMA trade blame over drug costs in House hearing

Pharmacy benefit manager lobby PCMA and drugmaker lobby PhRMA pointed fingers over problems in the prescription supply chain during the House committee's second PBM hearing on Tuesday.

By Rebecca Pifer • Sept. 19, 2023

De-risking visible particles through component selection

The visual inspection of injectable drug product has been a regulatory requirement since 1936, however, successful implementation of the standard has been challenging for many organizations.

By John Rech • Sept. 18, 2023

In the renal space, innovation could tackle health disparities. But the system doesn’t reward it.

Reimbursement challenges have created barriers for new dialysis products, according to the chief commercial officer of CorMedix.

By Michael Gibney • Sept. 14, 2023

A healthcare paradox: Drugs are too expensive but ‘easy’ to afford, Americans say

A new poll shows that Americans generally support several key IRA provisions, including Medicare price negotiations.

By Alexandra Pecci • Sept. 12, 2023

Ex-Sanofi executive Sibold joins Madrigal as new CEO

Bill Sibold has led Sanofi's specialty care business unit for the past six years. He replaces Paul Friedman as head of the NASH drug developer.

By Ned Pagliarulo • Sept. 11, 2023

Lawmakers are already gunning for more dramatic drug pricing reforms

Medicare just announced its first-ever drug price negotiations and legislators are quickly pushing to broaden the program.

By Karissa Waddick • Aug. 31, 2023

Finding the right flu vaccine is hard. Getting people to take it is harder.

Experts have determined the strains of this year’s flu season — now the question is whether people are too fatigued by the pandemic to be immunized.

By Michael Gibney • Aug. 30, 2023

Medicare named the first 10 drugs up for negotiation. Now what?

The list of blockbuster cardiovascular, diabetes and cancer drugs gives the industry a window into how regulators are approaching price negotiations.

By Karissa Waddick • Aug. 30, 2023

Drug shortages: The ‘fundamental problem’ that has pharma’s top regulator ‘fired up’

FDA Commissioner Robert Califf suggested larger reforms are needed to bolster the generic drug industry and prevent future medication shortages. 

By Karissa Waddick • Aug. 25, 2023

‘Eris’ and other new COVID strains accelerate vaccine push from Pfizer, Moderna, Novavax

New variants drive a need for another round of shots, and biopharma companies are stepping up to the plate.

By Michael Gibney • Aug. 24, 2023

A rare disease researcher aims to tackle disability discrimination in new lawsuit

How a pediatric neurologist is advocating for herself and the disabled community in a legal battle with the Howard Hughes Medical Institute.

By Alexandra Pecci • Aug. 22, 2023

A leading Democrat reveals how his mental health struggles shape his policy approach

Seattle Congressman Adam Smith's journey with anxiety and chronic health has influenced his perspective on healthcare and pharmaceutical policy.

By Karissa Waddick • Aug. 17, 2023

Pharma’s rare disease rush

Biogen’s acquisition of Reata Pharmaceuticals is the latest in a string of high-dollar rare disease deals over the last few months. What does it mean for industry?

By Meagan Parrish , Karissa Waddick • Aug. 14, 2023

PBMs could soon feel the regulatory sting

From Congress to the FTC to state legislatures, regulators are cracking the whip against PBMs.

By Karissa Waddick • Aug. 4, 2023

Big Pharma CEOs get candid about pricing and M&A

Head honchos at the biggest pharma companies reporting second-quarter earnings dig into the topics du jour.

By Michael Gibney • July 28, 2023

Biosimilar makers split strategies in bid to take on top-selling Humira

In challenging AbbVie for share of a $19 billion drug market, competitors are testing whether high upfront discounts or behind-the-scenes rebates can win them an advantage.

By Jonathan Gardner • July 26, 2023

With OTC birth control approved, access battles have just begun

Perrigo received the first FDA approval for an OTC birth control pill. Now, its global VP of women’s health says the company is working on the next challenge — affordability.  

By Karissa Waddick • July 26, 2023

For Merck, understanding maternal mortality is the first step toward equity

Merck has invested for more than a decade in reducing maternal death rates in the U.S. and globally. Now for the next chapter.

By Michael Gibney • July 25, 2023