100s of titles, one news app for just $10 a month.
Dive Deeper:
The Daily Biotech Pulse: Bristol Myers, BridgeBio Ink Oncology Pact, FDA Nod For Lava Therapeutics Blood Cancer Study And More
Here's a roundup of top developments in the biotech space over the last 24 hours:
23 Stocks Moving in Friday's Pre-Market Session
Gainers OTR Acquisition Corp. (NASDAQ: OTRA) rose 88.7% to $18.21 in pre-market trading after filing on Thursday showed shareholders approved…
56 Stocks Moving In Friday's Mid-Day Session
Gainers Veru Inc. (NASDAQ: VERU) shares jumped 62.5% to $14.67 after the company reported quarterly results on Thursday. Jefferies maintained…
The Daily Biotech Pulse: FDA Orphan Drug Tags For Neurocrine, Editas Candidates; Sale of BridgeBio's PRV; RMAT Designation For PolarityTE's Product
Here's a roundup of top developments in the biotech space over the last 24 hours:
One subscription that gives you access to news from hundreds of sites
90 Biggest Movers From Yesterday
Gainers Allena Pharmaceuticals, Inc. (NASDAQ: ALNA) surged 70.5% to close at $0.1330 on Thursday after declining 9% on Wednesday. Allena…
FDA Approves Lilly and Incyte's OLUMIANT (baricitinib) for Hospitalized Patients with COVID-19
The U.S. Food and Drug Administration (FDA) has approved OLUMIANT® (baricitinib) for the treatment of COVID-19 in hospitalized patients.
Get all your news in one place
Latest Technology news:
Push to make Victoria’s consent-free digital health database opt-in
The Victorian government’s plan for a centralised health database for all people with no consent has been labelled “bureaucratic imperialism”…
Read news from The Economist, FT, Bloomberg and more, with one subscription
Learn More
Jony Ive had one wacky idea for Apple's mixed reality headset
A new report details Apple's internal conflicts over its long-rumored mixed reality headset and the odd features it might ship…
Genesis GV60 To Come With 3 Years Of Free Charging On The Electrify America Network
We examine the announcement that Electrify America and Genesis have partnered to offer three years of unlimited charging for Genesis…
Facebook fails to stop Spanish language anti-abortion disinformation, study says
A group examined nine profiles reaching more than 2.79 million people peddling inaccurate information without consequence
Watch Ford's F-150 Lightning Tow A Trailer: How Far Can It Go?
Now that the Ford F-150 Lightning is shipping, we'll get to learn so much more about it from owners and…
From analysis to good news, read the world’s best news in one place
AWD-Only 2023 Genesis GV60 Is On Sale In The US, Starts At $58,890
The GV60 is offered in two packages in the United States, GV60 Advanced and GV60 Performance; both feature dual-motor AWD…
Why the Internet Hates Amber Heard
Notes on the paranoid style in online fandom

Stealth BioTherapeutics Shares Rally following FDA Orphan Drug Designation To Elamipretide For Duchenne muscular dystrophy (DMD)

By Ragothaman Srinivasan

Stealth BioTherapeutics Corp (NASDAQ:MITO) received Orphan Drug Designation to Elamipretide from the United States Food and Drug Administration (FDA) for the treatment of patients with Duchenne muscular dystrophy (DMD).

Reenie McCarthy, Chief Executive Officer, said, ”We are pleased that the FDA has recognized the high unmet need for innovative treatments for DMD, We look forward to further discussions with the FDA regarding our development initiatives, which we hope will bring new options to patients suffering from this devastating disease.”

Stealth Bio's Pre-IND meeting request was granted by FDA's Division of Neurology I, to discuss a development path for elamipretide in combination with products within the approved therapeutic class of exon-skipping phosphorodiamidate morpholino oligomers (PMO). 

Duchenne muscular dystrophy (DMD) is a fatal disease of muscle membrane instability in which the loss of dystrophin and ensuing mitochondrial dysfunction results in a cascade of events leading to progressive loss of muscle function.

FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Stealth BioTherapeutics shares are trading high 47 percent at $0.28 in the pre-market session.

What is inkl?
The world’s most important news, from 100+ trusted global sources, in one place.
Morning Edition
Your daily
news overview

Morning Edition ensures you start your day well informed.

No paywalls, no clickbait, no ads
Enjoy beautiful reading

Content is only half the story. The world's best news experience is free from distraction: ad-free, clickbait-free, and beautifully designed.

Expert Curation
The news you need to know

Stories are ranked by proprietary algorithms based on importance and curated by real news journalists to ensure that you receive the most important stories as they break.

Dive Deeper:
The Daily Biotech Pulse: Bristol Myers, BridgeBio Ink Oncology Pact, FDA Nod For Lava Therapeutics Blood Cancer Study And More
Here's a roundup of top developments in the biotech space over the last 24 hours:
23 Stocks Moving in Friday's Pre-Market Session
Gainers OTR Acquisition Corp. (NASDAQ: OTRA) rose 88.7% to $18.21 in pre-market trading after filing on Thursday showed shareholders approved…
56 Stocks Moving In Friday's Mid-Day Session
Gainers Veru Inc. (NASDAQ: VERU) shares jumped 62.5% to $14.67 after the company reported quarterly results on Thursday. Jefferies maintained…
The Daily Biotech Pulse: FDA Orphan Drug Tags For Neurocrine, Editas Candidates; Sale of BridgeBio's PRV; RMAT Designation For PolarityTE's Product
Here's a roundup of top developments in the biotech space over the last 24 hours:
One subscription that gives you access to news from hundreds of sites
90 Biggest Movers From Yesterday
Gainers Allena Pharmaceuticals, Inc. (NASDAQ: ALNA) surged 70.5% to close at $0.1330 on Thursday after declining 9% on Wednesday. Allena…
FDA Approves Lilly and Incyte's OLUMIANT (baricitinib) for Hospitalized Patients with COVID-19
The U.S. Food and Drug Administration (FDA) has approved OLUMIANT® (baricitinib) for the treatment of COVID-19 in hospitalized patients.
Get all your news in one place