| Date | Title | Description |
| 15.09.2026 | Gaucher Disease Drugs Market to Reach $4.3 Billion by 2036 as Long-Term Treatment and New Type 3 Therapies Reshape Rare-Disease Care | Enzyme replacement therapy leads with 58% share while Type 1 Gaucher disease accounts for 67% of 2026 demand
ROCKVILLE, Md., September 15, 2026 — The Gaucher disease treatment landscape is entering a new phase as established enzyme replacem... |
| 19.08.2026 | BioMarin to acquire Alesta Therapeutics for $275 million for potential rare bone disease treatment | BioMarin will acquire Alesta Therapeutics for $275 million upfront, and up to an additional $215 million based on milestones.
Alesta Therapeutic’s lead drug is a potential treatment for hypophosphatasia (HPP).
The deal bolsters BioMarin’s u... |
| 17.07.2026 | Eli Lilly to acquire BIOG portfolio company, AtaiBeckley Inc. | Eli Lilly to acquire BIOG portfolio company, AtaiBeckley Inc.
Fri, Jul 17, 2026 08:00 CET Report this content
17 July 2026
THE BIOTECH GROWTH TRUST PLC
(“BIOG” or the "Company")
Eli Lilly to acquire BIOG portfolio company, AtaiBec... |
| 16.06.2026 | DIMERIX EXCLUSIVELY LICENSES DMX-200 IN GREATER CHINA, SOUTH KOREA AND SOUTHEAST ASIA TO EVEREST MEDICINES | Everest Medicines licenses exclusive rights to commercialise DMX-200 for the treatment of Focal Segmental Glomerulosclerosis (FSGS) in Greater China, South Korea and Southeast Asia
500,000-1 million people are estimated to be living with FS... |
| 14.03.2024 | Rare Disease Advocates Learn to Accelerate Therapeutic Development at 9th RARE Drug Development Symposium | RARE Drug Development Symposium
The RARE Drug Development Symposium, hosted by Global Genes and the Orphan Disease Center of the University of Pennsylvania, equips advocates with the knowledge, skills and connections they need to advance th... |
| 05.12.2023 | Carmot’s chance of beating the odds in the biotech market just ended | Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.
Good morning, everyone. Damian here with a special thanks to everyone who joined us for STAT’s live event in New Yor... |
| 02.03.2022 | Alliance for Artificial Intelligence in Healthcare Elects New Board Leadership for 2022 | “AI will continue to have a demonstrable impact on human health. Having spent the last three years ideating with my fellow charter members to establish AAIH, I’m honored to take the reins and thrilled I get to engage with an incredible team... |
| 22.02.2022 | Tenth Annual Lake Nona Impact Forum Convenes Health And Wellbeing Thought Leaders In Orlando, Florida | Deepak Chopra speaks during the Lake Nona Impact Forum “There’s no doubt we live in a different world since the arrival of COVID-19,” said Impact Forum Co-Founder Gloria Caulfield. “This year’s Impact Forum is critical as we gather some of ... |
| 10.01.2022 | Amicus Therapeutics : Reports Preliminary 2021 Revenue and Provides 2022 Strategic Outlook and Revenue Guidance - Form 8-K | Amicus Therapeutics Reports Preliminary 2021 Revenue and Provides 2022 Strategic Outlook and Revenue Guidance
Full-Year 2021 Galafold® Revenue of ~$306M, Representing 17% YoY Growth
Expect Double-Digit Growth (15-20%) of 2022 Galafold Reven... |
| 22.11.2021 | Amicus Therapeutics : Long-term efficacy with migalastat in ERT-naïve and ERT-experience patients | Long-term multisystemic efficacy with migalastat in ERT-naïve and ERT-experienced patients with amenable GLA variants
Drago Bratkovic,1 Michel Tchan,2 Kathy Nicholls,3 Robert Hopkin,4 Gere Sunder-Plassmann,5 Derralynn Hughes,6 Eva Krusinska... |
| 09.11.2021 | Amicus Therapeutics Announces Third Quarter 2021 Financial Results and Corporate Updates | 3Q21 Total Galafold® Revenue of $79.5M – an 18% increase over 3Q20
Reiterating 2021 Revenue Guidance of $300M-$315M
AT-GAA BLA and NDA for Pompe Disease Accepted for Review by the U.S. FDA;
Marketing Authorization Applications Submitted to ... |
| 09.11.2021 | Amicus Therapeutics Announces Third Quarter 2021 Financial Results and Corporate Updates - Form 8-K | Amicus Therapeutics Announces Third Quarter 2021 Financial Results and Corporate Updates
3Q21 Total Galafold® Revenue of $79.5M - an 18% increase over 3Q20
Reiterating 2021 Revenue Guidance of $300M-$315M
AT-GAA BLA and NDA for Pompe Diseas... |
| 16.09.2021 | Poster: Cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study subgroup analyses | EP.196 Cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study subgroup analyses
Benedikt Schoser,1 Drago Bratkovic,2 Barry Byrne,3 Jordi Díaz-Manera,4 Pascal Laforet,5 Tahseen Mozaffar,6 A... |
| 05.08.2021 | Amicus Therapeutics : Announces Second Quarter 2021 Financial Results and Corporate Updates | 2Q21 Total Galafold® (migalastat) Revenue of $77.4M, a 24% increase over 2Q20
On-Track to Achieve Revenue Guidance of $300M-$315M
Completed the Rolling BLA and NDA Submissions to the U.S. FDA for AT-GAA in Pompe Disease
Positive EMA Rapport... |
| 02.08.2021 | Amicus Therapeutics : Announces European Commission Approval of Galafold® (migalastat) for Adolescents with Fabry Disease | PHILADELPHIA, Aug. 02, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced the Eu... |
| 21.05.2021 | AMICUS THERAPEUTICS, INC.
Poster: Phase 3 PROPEL Study of AT-GAA | Efficacy and safety of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo
in late-onset Pompe disease: PROPEL study
Benedikt Schoser,1 Drago Bratkovic,2 Barry Byrne,3 Jordi Diaz-Manera,4 Pascal Laforet,5 Tahseen Mozaffar,6 Ans... |
| 07.04.2021 | Plantronics : Poly Innovations Support a More Inclusive Workforce | As the world only just begins to see light at the end of the tunnel of the most horrific pandemic in a century, it is important that we pause to appreciate the few silver linings that came out of the experience. Surely, the ability to devel... |
| 07.04.2021 | PLANTRONICS, INC.
Plantronics : Poly Innovations Support a More Inclusive Workforce | As the world only just begins to see light at the end of the tunnel of the most horrific pandemic in a century, it is important that we pause to appreciate the few silver linings that came out of the experience. Surely, the ability to devel... |
| 12.02.2021 | Presentation: Topline Phase 3 PROPEL Results | Top Line Results From the PROPEL Phase 3 Study
Comparing AT-GAA (cipaglucosidase alfa/miglustat) Versus alglucosidase alfa/placebo In Late Onset Pompe
Disease
Benedikt Schoser
Friedrich-Baur-Institut
Neurologische Klinik und Poliklinik
LMU ... |
| 02.07.2019 | Gottlieb rebuts Science report on plummeting FDA inspections; Libtayo wins EMA and NICE endorsement | → The journal Science on Tuesday published a report that concluded the FDA’s compliance and enforcement actions have plummeted since Donald Trump took office, using data compiled from the agency’s own public records.
In ... |
| 15.12.2017 | Amicus CEO lobbied FDA to reverse FDA’s rejection of migalastat, claiming a delay of up to 7 years | Handwritten Note from John Crowley to Scott Gottlieb.
Five days after Scott Gottlieb was sworn in as head of the FDA last May, Amicus $FOLD CEO John Crowley was personally lobbying him to reverse a stinging rejection of mi... |
| 13.10.2017 | Biotech entrepreneur Saurabh Saha moves to translational research chief at Bristol-Myers; Ex-GSK pharma chief Hussain switches to private equity | Saurabh Saha
→ Bristol-Myers Squibb $BMY has hired Saurabh Saha to run their translation science group, which is responsible for triaging preclinical work of interest and steering the most promising programs toward the c... |
| 01.03.2017 | Trump signals elimination of ‘burdensome’ drug approval system | In a typically fiery speech, President Donald Trump in his first joint address to Congress last night tore into the FDA’s “slow and burdensome approval process,” and gave vague assurances that this would be “eliminated … so more lives could... |
| 29.11.2016 | Amicus maps out a 3-year delay at the FDA for lead drug, sending shares into a tailspin | CEO John Crowley
More than a year after Amicus Therapeutics was forced to do a sudden about-face and walk back promises of a quick submission for its lead drug migalastat, the biotech has laid out plans for a new trial that w... |
| 06.07.2016 | Stalled at the FDA, Amicus adds a preclinical rare disease drug in back-ended $90M deal | Amicus Therapeutics $FOLD has snagged a preclinical drug in a small biotech buyout, beefing up its slim pipeline at a time that the company is still assessing just how badly its lead drug may have stalled in front of the FDA.... |
| 31.05.2016 | Morning Read: Jazz Pharma makes $1.5B offer for Celator for acute myeloid leukemia drug | SV Life Sciences has raised $274 million in what will be its sixth fund to invest in life sciences startups. — Boston Business Journal
Amicus Therapeutics received a thumb’s up from the European Commission for its Fabry disease therapeutic ... |
| - | Morning Read: Jazz Pharma makes $1.5B offer for Celator for acute myeloid leukemia drug | TOP STORIES
Jazz Pharmaceuticals has made a $1.5 billion offer for Celator to gain access to its drug Vyxeos, which is used to treat acute myeloid leukemia. Earlier this month, the U.S. Food and Drug Administration granted breakthrough ther... |
| - | Self-made millionaire: Smart risk-takers don’t try to ‘change the world’—here’s what they do instead | Oftentimes, commencement speakers conclude their speeches with something to the effect of, “now go out and change the world.” Turns out, that may not be the best advice, according to a self-made millionaire.
Taking smart risks can ultimatel... |
