Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

quote:

Theo3 schreef op 12 juli 2021 11:14:

Pil of Spuit lijkt me toch een heel verschil Declan

quote:

Declan schreef op 12 juli 2021 11:10:

[...]
Is toch van geen enkel belang hier op te reageren.
Doorbraken is nog bij 30% minstens en dan nog de vraag hoe zwaar was de categorie HAE patiënten?
Overigens voor nu een net resultaat zeker voor patiënten .

Ruud..

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Declan schreef op 12 juli 2021 10:54:

[...]

Pharming wil 1000% zekerheid eerder komt het niet.
En ja het is nabij.

Ruud..

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Theo3 schreef op 12 juli 2021 11:14:

Pil of Spuit lijkt me toch een heel verschil Declan

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VriendjeStokVis schreef op 12 juli 2021 11:33:

[...]

Heb je ook niet een klein beetje het gevoel dat ze alles proberen in de doofpot te steken? Soon very soon is ondertussen meer dan een jaar. En bij iedere vraag voor update wordt Sijmen zenuwachtig.
Ze worden echt overal ingehaald en bouwen een steeds grotere bunker voor me gevoel.
Dat 10000% zeker willen zijn zeggen we nu al zo lang dat ik dat geloof beetje verloren ben..

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zjeeraar schreef op 12 juli 2021 12:15:

Het gilde heeft de koers geheel uitgekleed en is zodoende tot stilstand gekomen, goed bezig mag ik wel zeggen....wat een vertoning....

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LL schreef op 12 juli 2021 12:18:

[...]

Vermoedt u manipulatie? Maak dan meteen melding van deze criminele activiteiten!!

www.afm.nl/nl-nl/consumenten/themas/p...

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Declan schreef op 12 juli 2021 08:09:

[...]

Jij kan helemaal niet weten wat een normaal mens al dan niet nodig heeft om onpasselijk te worden.

Ruud..

quote:

LL schreef op 12 juli 2021 10:41:

BioCryst Reports 96-week Data from APeX-2 Showing ORLADEYO®(berotralstat) Reduced HAE Attack Rate by 80 Percent from Baseline

BioCryst Pharmaceuticals, Inc.
Sat, July 10, 2021, 4:00 PM

—Multiple data presentations at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2021 highlight durability of effect and long-term safety of ORLADEYO—

RESEARCH TRIANGLE PARK, N.C., July 10, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that hereditary angioedema (HAE) patients who were randomized to receive 150 mg of oral, once-daily ORLADEYO® (berotralstat) at the start of the APeX-2 trial had an 80 percent average reduction in their mean attack rate per month during weeks 25-96 of the trial, compared to baseline. Median attack rates also decreased from 2.7 attacks/month at baseline to 0.0 attacks per month in 16 of 17 months through the same period.

ORLADEYO was generally well-tolerated during the treatment period with fewer drug-related adverse events reported in part 3 (weeks 49-96) as compared to part 1 (weeks 0-24) and part 2 (weeks 25-48). Eighty-one percent of the patients who entered part 3 completed the trial.

“The long-term data we now see from two years of therapy in the clinical program reinforces the substantial, sustained reduction in HAE attacks patients experienced with ORLADEYO. These results are consistent with the experience many patients are having in the real world since we launched ORLADEYO, which is a key driver for the strong patient demand we are seeing as patients switch from injectable prophylactic agents and injectable acute-only therapies to oral, once-daily ORLADEYO for control of their HAE attacks,” said Dr. William Sheridan, chief medical officer of BioCryst.

The data are scheduled to be presented in an oral presentation on Monday, July 12, 2021 at 2:15pm CET at the EAACI Hybrid Congress 2021.

Additional Presentations of New ORLADEYO Data at EAACI

On-demand medication use was reduced in HAE patients treated with ORLADEYO (150 mg) in APeX-2

In HAE patients taking oral, once-daily ORLADEYO 150 mg who had a =50 percent reduction in their rate of investigator-confirmed attacks relative to their baseline attack rate, there was a 78 percent reduction in the use of on-demand medication (doses/month) from baseline to week 24, leading to 2.1 fewer doses of on-demand medication per month. In patients who had a =70 percent reduction in their rate of investigator-confirmed attacks relative to their baseline attack rate, there was an 85 percent reduction in the use of on-demand medication (doses/month) from baseline to week 24, leading to 2.2 fewer doses of on-demand medication per month.

The data were presented in poster #250 at the EAACI Hybrid Congress 2021.

ORLADEYO demonstrated consistently low attack rates in adolescent patients in APeX-S

In an analysis of adolescent patients (ages 12-17) treated with oral, once-daily ORLADEYO 150 mg in the open-label safety study, APeX-S, the mean (SEM) attack rate at week 4 was 0.4 attacks/month, which was generally sustained through week 48.

Median attack rates in these adolescents were 0.0 attacks/month throughout the 48 weeks of treatment.

Greater than 70 percent of patients were attack-free in weeks 4 to 48.

ORLADEYO was generally well-tolerated in APeX-S.

The data were presented in poster #336 at the EAACI Hybrid Congress 2021.

ORLADEYO demonstrated consistently low HAE attack rates during COVID-19

Stress is a documented trigger for HAE attacks and recently published physician and patient survey data shows an increase in patient-reported HAE attack rates due to an increase in stress related to the COVID-19 pandemic (pre-COVID: 1.5 attacks/3-months vs during COVID: 4.4 attacks/3-months).1

In an analysis of HAE patients taking oral, once-daily ORLADEYO 150 mg in the APeX-S trial, monthly HAE attack rates, pre-COVID and during COVID, remained consistently low, <1 attack/month, for patients treated with ORLADEYO. Patients receiving ORLADEYO maintained low attack rates during this time of high societal stress and disruption.

The data were presented in poster #166 at the EAACI Hybrid Congress 2021.

About ORLADEYO® (berotralstat)

ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

U.S. Indication and Important Safety Information

INDICATION
ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

Limitations of use
The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

The most common adverse reactions (=10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).

Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established.

There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

bron:
finance.yahoo.com/news/biocryst-repor...

quote:

Roberto schreef op 12 juli 2021 13:08:

Zijn er geen kopers of verkopen ze niet?

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zjeeraar schreef op 12 juli 2021 13:05:

0,962
12-jul-2021 13:00:43
Verschil
-0,001 (-0,10%)
Dagrange 0,955 - 0,965
Volume
471.968

Er valt ook voor het gilde geen droog brood meet te sturen....de rek is er wel uit........mooi zo......

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Ixalan68 schreef op 12 juli 2021 13:11:

[...]

Staat hier ook in hoeveel patiënten er zijn behandeld, ik zie het niet of lees ik erover heen?
Dat is nl. altijd wat van Pharming wordt gevraagd, aantallen en liefst veel.
Procenten zeggen me niet zoveel.
Bijwerkingen: buikpijn, overgeven, diarree, rugpijn en gasvorming ziektes.

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BassieNL schreef op 12 juli 2021 13:28:

[...]
Staat er niet in het artikel, maar dit is een linkje naar trial:

clinicaltrials.gov/ct2/show/NCT03485911

121 patiënten.. verdeeld over 2 groepen.

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Ixalan68 schreef op 12 juli 2021 13:30:

[...]

Dank je wel.
En hoeveel patiënten waren er nodig voor de toelating van Ruconest destijds?

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BassieNL schreef op 12 juli 2021 13:33:

[...]
Geen idee.. ga ik op niet opzoeken.