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    NEWS

    DRUGS & DEVELOPMENTS

    MARKET INTEL by PharmaCompass

    Egis is a Hungarian generic pharma company with 110 years history. Our activities incorporate all areas of the pharma value chain.
    All News #Library
    Rare Diseases

    VAXIL’s Annual and Special Shareholders Meeting

    GLOBENEWSWIRE

    Fortress Biotech Reports 2024 Financial Results and Highlights

    GLOBENEWSWIRE

    Palvella Therapeutics Reports 2024 Financial Results and Update

    GLOBENEWSWIRE

    Sobi publishes Annual and sustainability report for 2024

    PR NEWSWIRE

    Kazia Therapeutics Sells Cantrixil IP and Trademarks for Cantrixil

    PR NEWSWIRE

    Sarepta Announces Inducement Grants Under Nasdaq LR 5635(c)(4)

    BUSINESSWIRE

    PANTHERx Rare Selected for Vykat XR Distribution

    PR NEWSWIRE

    Scinai signs option agreement to acquire rare disease company Pincell

    PR NEWSWIRE

    Relmada Therapeutics Reports Q4 and Full Year 2024 Results

    GLOBENEWSWIRE

    Crinetics Pharma`s MAA, ODD For Paltusotine In Acromegaly Validated

    GLOBENEWSWIRE

    US FDA approves first treatment for Prader-Willi syndrome

    REUTERS

    Soleno Jumps As Analysts Hail Rare Disease Drug Approval

    REUTERS

    Savara Reports Q4 2024 Financial Results and Business Update

    BUSINESSWIRE

    Nkarta Reports 2024 Financial Results and Highlights

    GLOBENEWSWIRE

    Rezolute Appoints Rare Disease Leader Erik Harris to Board

    GLOBENEWSWIRE

    Mabwell to Present 6 studies at 2025 AACR Annual Meeting

    PR NEWSWIRE

    Eisai presents Leqembi® sales simulation at annual conference

    PR NEWSWIRE

    ReCode Therapeutics gets FDA Orphan Drug Designation for RCT2100

    BUSINESSWIRE

    X4 Pharmaceuticals Reports Q4 and Full Year 2024 Financial Result

    GLOBENEWSWIRE

    Palatin’s MC4R Agonist PL7737 Gets FDA Orphan Status For Obesity

    PR NEWSWIRE

    AccurEdit Therapeutics` ART001 Obtains FDA Orphan Drug Designation

    PR NEWSWIRE

    Rare variants in HECTD2 and AKAP11 increase bipolar disorder risk

    PR NEWSWIRE

    Santhera to update on AGAMREE® rollout at Capital Markets Day

    GLOBENEWSWIRE

    Pantherx Rare: Upsher-Smith Picks It For Vigafyde Oral Solution

    PR NEWSWIRE

    Precision AQ Expands AET to Tackle Rare Disease Challenges

    PR NEWSWIRE

    Novartis receives third FDA approval for oral Fabhalta

    GLOBENEWSWIRE

    Rhythm Pharmaceuticals Reacquires Imcivree® Rights in China

    GLOBENEWSWIRE

    BioInvent`s BI-1808 antibody featured at T-Cell Lymphoma Forum

    ACCESSWIRE

    iOnctura starts Phase II study in metastatic uveal melanoma

    PR NEWSWIRE

    Menarini partners VisualDx to Identify Blastic Plasmacytoid Cell BPDCN

    GLOBENEWSWIRE

    Alligator Gets FDA Orphan Drug Tag For HLX22 In Gastric Cancer

    ACCESSWIRE

    BioInvent gets FDA Orphan Drug Designation for BI-1808 for T-cell

    ACCESSWIRE

    Periodic Paralysis Association Unites Global Community

    BUSINESSWIRE

    Rhythm Pharma Gets Orphan Drug Status For Setmelanotide In Japan

    GLOBENEWSWIRE

    FIGHT DMD Trial Results Chosen for MDA Conference Presentation

    PR NEWSWIRE

    Henlius gets Orphan Designation for anti-HER2 mAb HLX22 in Cancer

    PR NEWSWIRE

    Can-Fite To Start Ph 2 Lowe Syndrome Trial With Piclidenoson

    GLOBENEWSWIRE

    AstraZeneca`s $800M rare disease bet hits primary Ph 3 goal

    FIERCE BIOTECH

    Savara to Present New Data From Phase 3 Trial at ATS 2025

    BUSINESSWIRE

    Capricor Therapeutic Show Long-Term Efficacy of Deramiocel for DMD

    GLOBENEWSWIRE

    PEP-Therapy gets Orphan Drug Designation for Pancreatic Cancer

    GLOBENEWSWIRE

    BioArctic receives Orphan Drug Designation for exidavnemab the US

    PR NEWSWIRE

    GRIN Therapeutics gets FDA Orphan Drug Designation for Radiprodil

    PR NEWSWIRE

    Nurix`s Bexobrutideg (NX-5948) Granted FDA Orphan Drug Designation

    GLOBENEWSWIRE

    KRRO-110 gets Orphan Designation for Alpha-1 Antitrypsin Deficiency

    GLOBENEWSWIRE

    Eton Pharmaceuticals Reports Positive Study Results for ET-600

    GLOBENEWSWIRE

    Cellectis Reports Financial Results for Q4 and Full Year 2024

    GLOBENEWSWIRE

    Ionis Sends Polycythemia Vera Drug to Ono in Deal Worth Nearly $1B

    BUSINESSWIRE

    PreventionGenetics to Manage Genetic Testing for Rare Diseases

    BUSINESSWIRE

    Atsena Wins FDA Fast Track For ATSN-201 Gene Therapy

    GLOBENEWSWIRE

    Oligomerix Publishes Results on Preclinical Study for Alzheimer`s

    BUSINESSWIRE

    Regeneron Science Talent Search 2025 Awards Over $1.8 Million

    GLOBENEWSWIRE

    Baylor Genetics to Showcase Innovations at ACMG 2025 Meeting

    GLOBENEWSWIRE

    Kaerus Bioscience Completes Phase 1 Trial for Fragile X Syndrome

    GLOBENEWSWIRE

    Longeveron Announces Nature Medicine Publication On Ph IIa Trial

    GLOBENEWSWIRE

    Lundbeck`s MSA treatment granted Orphan Drug Designation in Japan

    PR NEWSWIRE

    Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq List

    BUSINESSWIRE

    GENFIT Announces Approval of Amendment to 2025 OCEANEs Terms

    GLOBENEWSWIRE

    Inozyme Reports FY24, Prioritizes ENPP1 Deficiency Program

    GLOBENEWSWIRE

    ENCell’s EN001 Gets FDA Orphan Drug Tag For Charcot-Marie-Tooth

    BUSINESSWIRE

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