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US FDA to consider Dizal’s first-in-class lung cancer drug

Shanghai-based Dizal has made a submission to the US Food and Drug Administration, seeking approval for sunvozertinib. 8 November 2024

J&J files with EMA and FDA for Darzalex in high-risk smouldering MM

US healthcare giant Johnson & Johnson today revealed it has submitted applications with the US Food and drug Administration (FDA) and European Medicines Agency (EMA) to expand the approved indications for its Darzalex (daratumumab) subcutaneous (SC) formulation for the treatment of high-risk smouldering multiple myeloma (SMM). 8 November 2024

FDA Rare Pediatric Disease status for AskBio’s AB-1003

Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024

Acadia Pharma sells priority review voucher for $150 million

USA-based Acadia Pharmaceuticals has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction. 6 November 2024

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FDA approves REMS, advancing new drug disposal option for opioid analgesics

The US Food and Drug Administration (FDA) has announced approval of a modification to the Opioid Analgesic Risk Evaluation and Mitigation Strategy (OA REMS) 6 November 2024

FDA accepts Ionis NDA for donidalorsen

The US Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, under development by Ionis Pharmaceuticals. 4 November 2024

Disc Medicine leaps as it gets positive FDA feedback

Disc Medicine today announced a successful End of Phase 2 with the US Food and Drug Administration (FDA) supporting the regulatory path forward, including potential for accelerated approval, for bitopertin in erythropoietic protoporphyria (EPP). 4 November 2024

FDA sets up Rare Diseases Innovation Hub

On Friday, the US Food and Drug Administration published the FDA Voices: “FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub,” by Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research (CDER) and Dr Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER). 4 November 2024

FDA Rare Pediatric Disease designation for BPGbio

The US Food and Drug Administration (FDA) has granted a Rare Pediatric disease designation for BPM31510T, an investigational treatment for epidermolysis (EB) from BPGbio. 4 November 2024

The week in pharma: action, reaction and insight – week to November 1

US pharma major AbbVie last week proposed $1.4 billion acquisition of US CNS drug developer Aliada Therapeutics. Last week saw a bunch of third quarter 2024 financials results, among which were Elil Lilly, AbbVie and Merck & Co. Also of note, Lexicon’s type I diabetes drug Zynquista faced a Food and Drug Administration (FDA) advisory committee review that resulted in largely negative vote against recommendation. 3 November 2024

FDA panel vote leaves Lexicon’s Zynquista hopes hanging by a thread

Texas-based biopharma Lexicon Pharmaceuticals was trading nearly 36% lower on Friday morning. 1 November 2024

Novartis’ Scemblix gains FDA approval in newly diagnosed CML

Novartis late yesterday revealed that Scemblix (asciminib) was granted accelerated approval by the US Food and Drug Administration (FDA) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). 30 October 2024

FDA approves Protega’s Roxybond

Privately-held US drugmaker Protega Pharmaceuticals says that the US Food and Drug Administration (FDA) has approved Roxybond (oxycodone hydrochloride) immediate-release (IR) CII 10mg tablet for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. 29 October 2024

New FDA approval for Pfizer’s RSV vaccine Abrysvo

The US Food and Drug Administration (FDA) yesterday approved Abrysvo (respiratory syncytial virus vaccine). 23 October 2024

Sangamo shares jump as FDA clears faster gene therapy pathway

Shares in Sangamo Therapeutics surged around 40% on Tuesday, following the announcement that the US Food and Drug Administration has granted the company a faster path to approval for its gene therapy ST-920 (isaralgagene civaparvovec) for Fabry disease. 23 October 2024

FDA slaps CRL on Camurus NDA for CAM2029

Shares of Swedish drugmaker Camurus fell almost 10% to 588.50 kroner yesterday after it revealed that the US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the new drug application (NDA) for CAM2029 (octreotide) extended-release injection for the treatment of patients with acromegaly. 23 October 2024

FDA approves new presentation of biosimilar drug Selarsdi

Iceland-based Alvotech and the US unit of Israel’s of Teva Pharmaceutical Industries today revealed that that the US Food and Drug Administration (FDA) has approved Selarsdi (ustekinumab-aekn) in a new presentation 22 October 2024

Gilead to withdraw Trodelvy indication in USA

Biotech firm Gilead Sciences has announced plans to voluntarily withdraw the US accelerated approval for Trodelvy (sacituzumab govitecan-hziy) for the treatment of adults with locally advanced or metastatic urothelial cancer who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. 21 October 2024

FDA approves Astellas’ Vyloy

The US Food and Drug Administration has approved Vyloy (zolbetuximab-clzb), a claudin 18.2 (CLDN18.2)-directed cytolytic antibody, with fluoropyrimidine and platinum-containing chemotherapy, from Japanese drug major Astellas Pharma. 19 October 2024

FDA puts partial clinical hold on gotistobart trial

The US Food and Drug Administration (FDA) has placed a partial clinical hold on the two-stage, open-label, randomized Phase III trial, PRESERVE-003 (NCT05671510). 18 October 2024

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Phase III trial restarts in race to develop DMD gene therapy

People with Duchenne muscular dystrophy (DMD) have been given renewed hope that an investigational gene therapy under development by Pfizer may yield results. 29 April 2022

FDA approves Mycovia's Vivjoa, first med for chronic yeast infection

The US Food and Drug Administration (FDA) approved Vivjoa (oteseconazole) capsules, an azole antifungal indicated to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are not of reproductive potential. 28 April 2022

Alexion buy starts to pay off for AstraZeneca, with new US nod

Now the first and only long-acting C5 complement inhibitor in gMG, Ultomiris (ravulizumab-cwvz), has gained an additional approval in the USA. 28 April 2022

Partners seek EUA for COVID-19 booster jab in kids aged five to 11

Pfizer and BioNTech have submitted an application to the US Food and Drug Administration (FDA) for Emergency Use Authorization (EUA) of a 10-µg booster dose of their COVID-19 jab for children aged five to 11 years of age. 27 April 2022

More breast cancer patients could benefit from treatment with Enhertu

An antibody-drug conjugate (ADC) developed by British drugmaker AstraZeneca and its Japanese partner Daiichi Sankyo has been granted Breakthrough Therapy designation (BTD) in the USA. 27 April 2022

FDA approves first COVID-19 treatment for young children

Yesterday, the US Food and Drug Administration expanded the approval of the COVID-19 treatment Veklury (remdesivir) to include pediatric patients 28 days of age and older weighing at least 3 kilograms (about 7 pounds) with positive results of direct SARS-CoV-2 viral testing, who are: hospitalized, or not hospitalized and have mild-to-moderate COVID-19 and are at high risk for progression to severe COVID-19, including hospitalization or death. 26 April 2022

Axsome expects CRL on migraine therapy AXS-07

Central nervous system (CNS) specialist Axsome Therapeutics revealed that, on April 22, it was informed by the US Food and Drug Administration that chemistry, manufacturing, and controls (CMC) issues identified during the FDA’s review of the company’s New Drug Application (NDA) for its AXS07 (meloxicam and rizatriptan) product candidate for the acute treatment of migraine are unresolved. 26 April 2022

FDA accepts tremelimumab for priority review

UK pharma major AstraZeneca’s Biologics License Application (BLA) for tremelimumab has been accepted for Priority Review in the USA, supporting the indication of a single priming dose of the anti-CTLA4 antibody added to Imfinzi (durvalumab) for the treatment of patients with unresectable hepatocellular carcinoma (HCC). 25 April 2022

Ampio Pharma tumbles as it faces FDA delay on osteoarthritis drug

Shares of Ampio Pharmaceuticals closed down 27% at $0.25 yesterday, as the US biopharma company announced the receipt of written responses from the US Food and Drug Administration regarding the AP-013 trial results, suggesting it will likely need to perform a further Phase III study. 22 April 2022

FDA 'Breakthrough' status for Zambon's CMS I-neb

Privately-held Italian drugmaker Zambo today announced that the US Food and Drug Administration has granted Breakthrough Therapy designation to colistimethate sodium powder for nebulization solution (CMS I-neb) for the reduction in the incidence of pulmonary exacerbations in adult patients with non-cystic fibrosis bronchiectasis (NCFB) colonized with P. aeruginosa. 21 April 2022

FDA grants RMAT designation to AlloVir's posoleucel

Shares of USA-based AlloVir shot up more than 46% to $7.90 in mid-morning trading, after it announced that the US Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational multi-virus-specific T cell therapy, posoleucel. 20 April 2022

Teva and MedinCell get CRL for TV-46000/mdc-IRM NDA

The US subsidiary of Teva Pharmaceutical Industries and MedinCell announced that the US Food and Drug Administration has issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for TV-46000/mdc-IRM (risperidone extended-release injectable suspension for subcutaneous use) for the treatment of schizophrenia. 20 April 2022

US FDA accepts NDA for daprodustat

UK pharma major GlaxoSmithKline today announced that the US Food and Drug Administration HAS accepted the New Drug Application (NDA) for daprodustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), for the potential treatment of patients with anemia of chronic kidney disease (CKD). 19 April 2022

Enhertu sBLA accepted for priority review by FDA

The US Food and Drug Administration has accepted the supplemental Biologics License Application (sBLA) for Enhertu (trastuzumab deruxtecan) for the treatment of adult patients in the USA with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 (ERBB2) mutation and who have received a prior systemic therapy. The application has also been granted Priority Review. 19 April 2022

The FDA's plans for pediatric therapeutics

The development of pediatric therapies faces numerous obstacles in the pharmaceutical industry. Among these obstacles are reluctance to expose children to potential harm from experimental therapies, fear of increased liability from pediatric studies, and lack of incentive because of the small market share represented by several pediatric medicines.1 19 April 2022

TG Therapeutics pulls BLA/sNDA for U2 in CLL and SLL and sale of Ukoniq

US biotech TG Therapeutics saw its shares plunge almost 32% to $6.03, after it announced the voluntary withdrawal of the pending Biologics License Application (BLA)/supplemental New Drug Application (sNDA) for the combination of ublituximab and Ukoniq (umbralisib) - combination referred to as U2 - for the treatment of adult patients with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). 19 April 2022

FDA guidance for industry on bioavailability studies submitted in NDAs or INDs

The US Food and Drug Administration has issued a final guidance to drug sponsors on conducting and submitting bioavailability (BA) studies for drug products in investigational new drug applications (INDs), new drug applications (NDAs), and NDA supplements. 19 April 2022

CMOs to keep prospering from approvals of COVID-19 products and cell therapies

The US Food and Drug Administration’s (FDA) increasing approvals and Emergency Use Authorizations (EUAs) of COVID-19 vaccines and drugs, as well as cell and gene therapies, continue to provide ample manufacturing opportunities for contract manufacturing organizations (CMOs), says GlobalData. 14 April 2022

FDA guidance on racial and ethnic diversity in clinical trials

On Wednesday, the US Food and Drug Administration issued a new draft guidance to industry for developing plans to enroll more participants from underrepresented racial and ethnic populations in the USA into clinical trials – expanding on the agency’s previous guidance for industry to improve clinical trial diversity. 14 April 2022

Thoughts on FDA's proposal to extend the FDR process to withdrawals of accelerated approvals

The US Food and Drug Administration recently transmitted its justification to Congress for its Fiscal Year 2023 budget. 13 April 2022

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